After over a decade of preclinical research and development, a new gene therapy treatment for sickle cell anemia (SCA) is reversing disease symptoms in two adults. Punam Malik, MD, is the principal investigator.
A daily hydroxyurea pill may bring relief for young children living with sickle cell anemia (SCA) in sub-Saharan Africa, says study in The New England Journal of Medicine led by Russell Ware, MD, PhD.
A new study, led by David Schnadower, MD, and published in The New England Journal of Medicine, shows that a commonly used probiotic is not effective in improving symptoms in young patients with gastroenteritis.
The TQL Foundation donated $1 million to benefit cancer research at the Proton Therapy Center--the only center in the world with a dedicated research gantry.
New technology analyzes linguistic and vocal characteristics of sessions between therapists or physicians, and patients to provide a near real-time assessment of a patient at risk for suicide or mental health conditions.
A new study of high school soccer players suggests a neck collar may help protect the brain from head impacts over the course of a competitive soccer season. The study was led by Greg Myer, PhD, and published in the British Journal of Sports Medicine.
Researchers report in PLoS Biology finding new clues about what controls the timing of birth and how these may lead to new answers for this persistent public health challenge.
Researchers led by Ashish Kumar, MD, PhD, report in Pediatric Blood & Cancer Physicians should not rush into HLH treatment without also checking for the possibility of cancer in patients with an initial HLH diagnosis.
Scientists, led by Yi Zheng, PhD, report in the journal Leukemia a new approach for bone marrow donation and transplant that could make the life-saving procedure safer and more effective for patients.
Scientists working to bioengineer the entire human gastrointestinal system in a laboratory now report in Cell Stem Cell using pluripotent stem cells to grow human esophageal organoids.
Researchers, led by Theresa Alenghat, VMD, PhD, report in JCI Insights finding an epigenetic signature in patient cells that appears to predict inflammation risk in a serious type of inflammatory bowel disease (IBD) called Crohn’s disease.
Overall well-being of a population on a county level is associated with lower healthcare spending for each Medicare fee-for-service beneficiary, says JAMA Network Open study led by Carley Riley, MD.
A new study in Science Translational Medicine could have significant implications for a growing field of study called circadian medicine—timing the administration of drugs or other therapies to coincide with the body’s internal clock.
Researchers report in Nature Communications that blocking an RNA-silencing protein in the livers of mice keeps the animals from getting fat and diabetic conditions.
Shari Wade, PhD, worked with the CDC and others to develop 19 clinical recommendations that cover diagnosis, prognosis, and management and treatment of concussion. The Pediatric mTBI Guideline was published in JAMA Pediatrics.
Researchers led by Gang Huang, PhD, report in Cancer Discovery identifying a gene that drives molecular processes leading to the diverse types of MDS disorders that affect people.
Researchers, led by Bruce Trapnell, MD, report in Nature Communications they figured out why air sacs in the lungs clog up with a thick substance called surfactant in a brutal disease called Pulmonary Alveolar Proteinosis (PAP).
Vitamin D supplementation may help prevent asthma if taken prenatally or given to children postnatally, according to a new study led by Cincinnati Children’s researchers and published in the Journal of Allergy and Clinical Immunology.
JAMA Pediatrics study led by Katherine Auger, MD, examines post-discharge education and reutilization rates.
Research suggests that a drug similar to one under evaluation as a possible cancer treatment might eventually help people born with fragile X syndrome (FXS). The study was published in Neuropsychopharmacology.
Scientists, led by Lee Grimes, PhD, report in the Journal of Experimental Medicine identifying a potential treatment target for an aggressive and deadly blood cancer called acute myeloid leukemia (AML).
Researchers, led by Q. Richard Lu, PhD, report in Developmental Cell that mutation or loss of a gene called CHD8 hinders formation of the sheath in developing oligodendrocytes, causing neurological defects in the animals.
Scientists, led by Biplab Dasgupta, PhD, and Rishi Raj Chhipa, PhD, report in Nature Cell Biology finding a potentially promising treatment target for aggressive and deadly high-grade brain cancers like glioblastoma.
A protein in the cell nucleus already targeted therapeutically for several types of cancer has now been linked to an aggressive form of pediatric liver cancer called hepatoblastoma (HBL), according to a study led by Nikolai Timchenko, PhD, and published in the Nature journal Communications Biology.
A new study led by Nurit Azouz, PhD, and Marc Rothenberg, MD, PhD, and published in Science Translational Medicine reveals that the absence of a specific protein in cells lining the esophagus may cause inflammation and tissue damage in people with eosinophilic esophagitis (EoE).
Researchers report in the journal iScience their data show that radio frequency identification (RFID) chips could be used to sense, record, and track interesting changes live in large quantities of organoids at once during testing for possible therapeutic use in patients.
A new study, led by Raphael Kopan, PhD, and published in PNAS, reveals potential mechanisms that could help preterm infants extend a crucial nephron development window.
Scientists, led by Ertugrul Ozbudak, PhD, report in Cell Reports new findings could uncover methods for modulating genetic signals to prevent birth defects or cancers rooted at the earliest stages of development.
Researchers tissue-engineered human pancreatic islets that secrete hormones like insulin and successfully treat sudden-onset type 1 diabetes in transplanted mice. The study was published in Cell Reports.
A pilot study, led by Cincinnati Children’s Drew Barzman, MD, indicates that artificial intelligence may be useful in predicting which students are at higher risk of perpetrating school violence. The study is published in the journal Psychiatric Quarterly.
Scientists led by Marc Rothenberg, MD, PhD, uncover three distinct subtypes of eosinophilic esophagitis (EoE), an emerging food allergic disease.
Scientists discover the mechanism behind an unusual form of tissue regeneration that may someday reduce the need for expensive and difficult-to-obtain organ transplants. Findings were published in the journal Nature.
Researchers, led by Yutaka Yoshida, PhD, report in Cell Reports the mapping of critical nerve connections to the spine that drive voluntary movement in forelimbs.
Scientists report in the journal Molecular Cancer Therapeutics using an experimental molecular therapy in preclinical laboratory tests to effectively treat several types of deadly pediatric brain cancer.
Cincinnati Children’s scientists report that the Epstein-Barr virus appears to participate in the development of lupus, multiple sclerosis, rheumatoid arthritis, juvenile idiopathic arthritis, inflammatory bowel disease, celiac disease, and type 1 diabetes.
In a study published by Circulation, researchers led by Burns Blaxall, PhD, demonstrate a novel therapeutic peptide stops the process and heart fibrosis in mice with heart injury and human heart failure cells.
Researchers led by Tanya Kalin, MD, PhD, report in Cell Reports that loss of the gene Foxf1 in lung myofibroblasts increases the severity of idiopathic pulmonary fibrosis (IPF) in mice.
Cincinnati Children’s researchers, led by Ted Folger, PhD, report in the journal Pediatrics a link between parents impacted by adverse childhood experiences and increased risk for delayed development of their children at age two.
Cincinnati Children’s is part of a national effort to prevent a global pandemic of the H7N9 strain of avian flu spreading with growing alarm in regions of China. Paul Spearman, MD, is the lead investigator of the study’s Cincinnati arm.
A study of more than 10,000 adults with congenital heart disease (CHD) shows an increased risk for early dementia in middle-age adults. The study, led by Nicolas Madsen, MD, was published in Circulation.
Researchers, led by Fumika Hamada, PhD, and Hitoshi Okamura, MD, PhD, report in Genes & Development the first molecular evidence that two anciently conversed proteins with shared biological ancestry regulate body temperature rhythms in insects and mammals.
Genomic profiling of mostly untreatable and deadly nerve sheath tumors led scientists to test a possible therapy that inhibited tumor growth in lab tests on human tumor cells and mouse models. The study, led by Q. Richard Lu, PhD, was published in Cancer Cell.
Scientists from Cincinnati Children’s test experimental molecular therapy in the mice that restores nerve insulation and improves limb function following nerve injury. The study, led by Q. Richard Lu, PhD, was published in Nature Medicine.
Cincinnati Children’s researchers used 3D imaging with molecular testing to uncover new insight into the earliest stages of mammalian pregnancy. The study was published in Nature Communications.
Scientists, led by Takanori Takebe, MD, publish data in Stem Cell Reports about a bioengineering strategy that uses induced pluripotent stem cells to generate posterior gut endodermal progenitor cells (PGECs).
Cincinnati Children’s researchers discover how gene mutation affects T cell function to promote immune disorders. Findings were published in Nature Communications.
A new study of infants born in withdrawal due to opioid exposure in utero shows a dramatic increase in torticollis - an abnormal twisting of the neck. The study, led by Jenny McAllister, MD, was published in The Journal of Pediatrics.
Women exposed to air pollution just before or after conception face an increased risk of their children being born with birth defects. Emily DeFranco, DO, is senior author of the study published online in The Journal of Pediatrics.
Researchers led by Takanori Takebe, MD, report in Cell Reports creating a biologically accurate mass-production platform that overcomes major barriers to bioengineering human liver tissues suitable for therapeutic transplant into people.
Researchers led by Jorge Bezerra, MD, report in Science Translational Medicine finding a strong biomarker candidate for early diagnosis of biliary atresia.
Research led by Sing Sing Way, MD, PhD, and published in PLoS Pathogens suggests women who develop symptom-free Zika infections build immunity that will protect them and their fetal offspring.
FDA approval “represents a breakthrough for the field and offers a sigh of relief for the many patients suffering from eosinophilic disorders,” says Marc Rothenberg, MD, PhD.
Medical center launches the Center for Stem Cell and Organoid Medicine (CuSTOM) to accelerate the transfer of stem cell-organoid technology from the research lab to practical clinical tool.
A new study led by Mary Greiner, MD, and published online in Pediatrics, examines routine laboratory screenings for children entering foster care.
Early research results in Nature Communications suggest scientists might be on to a way to preserve cardiac function after heart attacks or for people with certain inherited heart defects. Katherine Yutzey, PhD, is the lead study author.
Researchers, led by Jareen Meinzen-Derr, PhD, say children who are deaf or hard of hearing have the capacity for higher language levels. The study is published online in the Journal of Developmental and Behavioral Pediatrics.
Researchers led by Satoshi Namekawa, PhD, report in Genes & Development identifying the key molecular and genetic switch that activates production of healthy male sperm.
Study based on data from more than 50,000 women, published in The New England Journal of Medicine and led by Drs. Muglia and Zhang, could lead to new ways to prevent leading cause of infant mortality.
A new study published in SLEEP supports calls for public health efforts to promote adolescent sleep, according to Dean Beebe, PhD, senior author of the study.
Cincinnati Children’s researchers report in the Journal of the American College of Cardiology encouraging preclinical results as they pursue elusive therapeutic strategies to repair scarred and poorly functioning heart tissues after cardiac injury.
Researchers led by Yutaka Yoshida, PhD, report in Science blocking molecular pruning of sophisticated brain-to-limb nerve connections in maturing mice.
A multi-institutional scientific team led by Matthew Flick, PhD, uncovers a previously unknown molecular link in the body that drives obesity and inflammation. The study was published in the Journal of Clinical Investigation.
A new study, led by Kevin Hommel, PhD, shows that patients who take their medication as prescribed have lower healthcare costs than those who do not. The study was published in Inflammatory Bowel Diseases, the journal of the Crohn’s & Colitis Foundation.
Scientists, led by James Wells, PhD, use human pluripotent stem cells (hPSCs) to generate human embryonic colons in a laboratory that function much like natural human tissues when transplanted into mice, according to research published in Cell Stem Cell.
An international team of researchers led by Takanori Takebe, MD, and colleagues at the Max Planck Institute in Germany, are bioengineering human liver tissues for their potential to study and treat liver disease. Results were reported in Nature.
Reporting results in Nature Communications, Cincinnati Children's researchers seek ways to develop regenerative therapies for muscle disorders by getting stem cells to fuse and form functioning skeletal muscle tissues.
A new study, publish in the journal PLOS ONE and led by Cincinnati Children's researchers, shows that engaging with children while reading them books gives their brain a cognitive boost.
Researchers led by Michael B. Jordan, MD, report they were able to selectively inhibit DNA damage repair in activated T cells as part of an experimental combination treatment for immune diseases in mouse models. The study was published in PNAS Early Edition.
Vaccinating pregnant mothers year-round against flu in subtropical Nepal reduced infant flu virus infection rates by an average of 30 percent and increased birth weights by 15 percent. The study was published in The Lancet Infectious Diseases.
Scientists led by Yutaka Yoshida, PhD, report in Neuron the lost function of two genes prevents infant laboratory mice from developing motor skills as they mature into adults.
Scientists led by Q. Richard Lu, PhD, write in Nature Communications it may be possible to therapeutically fine tune a constantly shifting balance of molecular signals to ensure the body’s peripheral nerves are properly insulated and functioning normally.
Researchers, led by Vladimir Kalinichenko, MD, PhD, report in Science Signaling that treatment with a new experimental molecular compound called RCM-1 prevents excess inflammation and mucous in mouse models and human respiratory cells.
Children may carry significant levels of nicotine on their hands even when parents are not smoking around them, according to a new study. The study, co-authored by Melinda Mahabee-Gittens, MD, was published in Tobacco Control.
Researchers led by Q. Richard Lu, PhD, report in Developmental Cell microRNA treatment partially repaired damaged nerves and restored limb function in mice with MS.
Reporting results in Nature Medicine, researchers led by Mohammad Azam, PhD, suggest that blocking two signaling proteins as part of combination therapy might cure several types of kinase-driven, treatment-resistant leukemia and solid tumor cancers.
Intestinal stem cells rejuvenate daily so bowels will stay healthy and function normally, but a new study in Cell Reports suggests they also age along with people and lose their regenerative capacity.
Scientists led by Senad Divanovic, PhD, identify a molecular driver of inflammation that may finally answer a key question about what causes mild systemic prenatal infections to trigger preterm birth. Findings were reported March 9 in The Journal of Clinical Investigation Insight (JCI Insight).
As people get older so do the hematopoietic stem cells (HSCs) that form their blood, creating an increased risk for compromised immunity and certain blood cancers. Researchers led by Hartmut Geiger, PhD, report in the The EMBO Journal that the bone marrow niche where HSC’s form also ages, contributing to the problem.
Scientists led by Cincinnati Children's Manoj Pandey, PhD, propose in Nature blocking a molecule that drives inflammation and organ damage in Gaucher and maybe other lysosomal storage diseases as a possible treatment with fewer risks and lower costs than current therapies.
Genomic testing of biopsies from patients with deadly, treatment-resistant cancerous blood syndromes allowed doctors to identify genes fueling the ailments and use targeted molecular drugs to successfully treat them. Researchers, led by Ashish Kumar, MD, PhD, report their data in Journal of Clinical Investigation Insight.
Cincinnati Children’s researchers uncover long-term effects of traumatic brain injury (TBI) - an average of seven years after injury. Brad Kurowski, MD, MS, presented findings during the annual meeting of the Association of Academic Physiatrists.
A new study published in Science Translational Medicine reveals that consequences of routine antibiotic use may be deeper and longer lasting than expected. “It is time to begin pushing back on practices that were established decades ago, when our level of understanding was different,” says lead author, Hitesh Deshmukh, MD, PhD.
Cincinnati Children’s scientists led by Jim Wells, PhD, report in Nature using pluripotent stem cells to generate human stomach tissues in a petri dish that produce acid and digestive enzymes.
Researchers led by Daniel Starczynowski, PhD, report in Nature Immunology a new mechanism that controls blood cell function and several possible molecular targets for treating myelodysplasia syndromes (MDS).
Years of laboratory research by the team of Cincinnati Children’s cancer biologist Nancy Ratner, PhD, has led to a new study in the New England Journal of Medicine showing targeted molecular therapy for NF1 plexiform neurofibromas helps children with the genetic disorder.
STEMCELL Technologies Inc. has signed an exclusive license agreement with Cincinnati Children’s to commercialize its fundamental technology for generating gastrointestinal organoids from pluripotent stem cells (PSCs).
Researchers have cracked the complete genetic code of individual cells in healthy and diseased human lung tissues to find potential new molecular targets for diagnosing and treating the lethal lung disease Idiopathic Pulmonary Fibrosis (IPF). Results are published in the Journal of Clinical Investigation Insights (JCI Insights).
Cincinnati Children’s Hospital Medical Center has appointed Andrew Wooten, MS, MBA, to the position of vice president of its Center for Technology Commercialization (CTC). Wooten will begin his position on Jan. 3, 2017.
Researchers, led by Michael Helmrath, MD, and Jim Wells, PhD, report in Nature Medicine using human pluripotent stem cells to grow human intestinal tissues that have functioning nerves in a laboratory, and then using these to recreate and study a severe intestinal nerve disorder called Hirschsprung’s disease.
According to a study published online in The New England Journal of Medicine, one of every four children admitted to pediatric intensive care units around the world develops acute kidney injury (AKI). Moreover, the nearly 12 percent who develop more severe AKI have a further increased risk of death within 28 days, according to lead author Stuart L. Goldstein, MD, director of the Center for Acute Care Nephrology at Cincinnati Children's.
A new study published by Pediatrics suggests that children recovering from complex pneumonia should use oral antibiotics instead of intravenous antibiotics. The retrospective study, led by Samir Shah, MD, MSCE, and director of Hospital Medicine at Cincinnati Children's, included 2,123 children at 36 hospitals.
A new study shows that computer technology known as machine learning can be a reliable tool for classifying people as suicidal. These results provide strong evidence for using advanced technology as a decision-support tool to help clinicians and caregivers identify and prevent suicidal behavior, says John Pestian, PhD, professor in the divisions of Biomedical Informatics and Psychiatry at Cincinnati Children’s and the study’s lead author.
A recently published study in The New England Journal of Medicine shows no significant differences among amitriptyline, topiramate and placebo in reducing headache days or related disability. Researchers, including Andrew Hershey, MD, PhD, and Scott Powers, PhD, conducted the 24-week clinical trial that included 328 eligible patients.
The U.S. Department of Health and Human Services’ Office of the National Coordinator for Health Information Technology (ONC) has awarded Keith Marsolo, PhD, a Standards Exploration Grant to study the cost efficiencies of integrating clinical research systems within a medical center’s electronic health record (EHR).
A team of Cincinnati Children's researchers, led by Satoshi Namekawa, PhD, identified a network of proteins often linked to cancer as also important to male fertility and the birth of healthy offspring, according to a study in the Oct. 18 online issue of Cell Reports.
Sing Sing Way, MD, PhD, Division of Infectious Diseases, is receiving the Pioneer Award from the National Institutes of Health. Part of NIH’s High Risk High Reward program, the Pioneer Award helps investigators pursue new research directions and develop groundbreaking, high-impact approaches to biomedical and behavioral science, according to NIH. The award totals $5.5 million over five years. The funding will help Way and his colleagues advance research into immunological mechanisms responsible for shifts in infection susceptibility in babies with the goal of more effective therapeutic and preventative strategies.
Groundbreaking research into how the immune system works in early newborn development has led to Cincinnati Children’s physician Sing Sing Way being named a Faculty Scholar by The Howard Hughes Medical Institute, the Simons Foundation and the Bill & Melinda Gates Foundation.
A recent study by Cincinnati Children’s researchers reveals increased self-injury and suicide attempt rates for transgender youth. “Our study provides further evidence for the at-risk nature of transgender youth and emphasizes that mental health providers and physicians working with this population need to be aware of these challenges,” says Claire Peterson, PhD, a psychologist at Cincinnati Children’s and lead author of the study. Findings are published in Suicide and Life-Threatening Behavior.
Findings published in Nature reveal that before a blood cell’s specific type is defined, it is caught in a tug of war by competing genetic signals. “It’s a basic finding that helps us address a fundamental question of developmental biology – what are the nature of the intermediate states and the networks of regulatory genes that underlie cell-type specification,” said Harinder Singh, PhD, study co-author and Director of Immunobiology and the Center for Systems Immunology at Cincinnati Children’s.
Researchers at Cincinnati Children’s Hospital Medical Center recently developed a way to assess middle and high school students’ risk of violent behavior at schools. The study included 25 students with behavioral changes from 15 schools in Ohio and Kentucky. The study results were published in July 2016 in Psychiatric Quarterly. The study, which was done over the course of one year, was a collaborative effort among Ohio and Kentucky schools, the Cincinnati Children’s forensic research team led by Drew H. Barzman, MD, and parents of the students.
A new Cincinnati Children’s Hospital Medical Center study shows that as many as 25 percent of preterm births might be attributed to abnormalities in the interval between pregnancies, the mother’s body mass index prior to pregnancy, and the amount of weight gain in pregnancy. All of these risk factors may be modifiable to reduce the risk of premature births – those before 37 weeks of gestation. The study is published online in the Maternal and Child Health Journal. Emily DeFranco, DO, a physician-researcher at the Center for Prevention of Preterm Birth and her colleagues conducted the study from birth records in Ohio from 2006 to 2011.
In study results published online in PNAS (Proceedings of the National Academy of Sciences), Cincinnati Children’s researchers report findings that open a path to earlier non-invasive diagnosis of heart complications in Sickle Cell Anemia (SCA) patients. Scientists, including senior author Punam Malik, MD, also report development of new targeted therapies to help SCA patients live longer with better quality of life.
Metformin, a medication commonly used for the treatment of type 2 diabetes, may also play an unexpected role in blocking a significant cause of preterm birth, according to findings published online in JCI: The Journal of Clinical Investigation. The early-stage study, based on results from mice bred to be prone to premature birth, was led by a team of scientists at Cincinnati Children’s, including senior author Sudhansu K. Dey, PhD, along with colleagues in France and Japan.
According to a multi-institutional study published online July 26 in Pediatrics, a new web-based software program is helping reduce ADHD behavioral symptoms in children receiving care at community pediatric practices. The software aids in coordinating care and ensuring patients get the most effective ADHD medications. “Our data show the software not only helped improve the quality of medication care received by children treated at community based pediatric practices, but it also improved treatment outcomes for these children,” said principal investigator Jeffery Epstein, PhD.
According to a new study, children born with heart disease have an increased risk of developing type 2 diabetes after age 30 – particularly those born with a cyanotic congenital heart disease (CHD) condition. "Given the cardiovascular health burden of type 2 diabetes, attention to its development in CHD survivors is warranted,” says Nicolas Madsen, MD, a cardiologist at Cincinnati Children’s and lead author of the study, published online in the Journal of the American Heart Association.
Some of the most effective treatments for autism spectrum disorder (ASD) are also well known for their risk of weight gain and subsequent health complications. For the first time, however, researchers, including Cincinnati Children’s Logan Wink, MD, have compared five of these second generation antipsychotics (SGAs) to determine which ones are the biggest culprits. Results are published online in the Journal of Child and Adolescent Psychopharmacology.
A new online open-access database has been developed to allow the clinical responses of more than 5 million patients to all FDA-approved drugs to be used to identify unexpected clinical harm, benefits and alternative treatment choices for individual patients, according to a study appearing July 8 in Nature Biotechnology. The database is the result of work by scientists in Cincinnati Children’s Division of Biomedical Informatics and the Center for Clinical and Translational Science Training.
In collaboration with Cradle Cincinnati, a Cincinnati Children’s study finds that many more women may be smoking and exposed to nicotine than previously thought. “This is extremely important new information for us as we work to better understand risk factors for preterm birth,” said Jim Greenberg, MD, director of the Perinatal Institute at Cincinnati Children’s and senior author of the study. Results are published in the Journal of Perinatology.
Research led by Greg Myer, PhD, director of sports medicine research at Cincinnati Children’s, shows promise for a novel device designed to protect athletes from sports-related brain injuries. Findings have been recently published in Frontiers in Neurology | Neurotrauma and in the British Journal of Sports Medicine.
Cincinnati Children’s researchers, report in the journal Cell Reports that a targeted molecular therapy dramatically reduces the initial development of Non Alcoholic Fatty Liver Disease (NAFLD) in laboratory mouse models. Nikolai Timchenko, PhD, is senior author and the head of Cincinnati Children’s Liver Tumor Biology Program.
Researchers at Cincinnati Children’s, including senior author Amy Sanghavi Shah, MD, from the Division of Endocrinology, have discovered that teenagers with type 2 diabetes have significant changes in total brain gray matter volume. Changes are also noted in regions of gray matter involved in seeing and hearing, memory, emotions, speech, decisions making, and self-control.
Researchers including Jeffery Molkentin, PhD from the Division of Molecular Cardiovascular Biology have developed an experimental model that uses genetics-guided biomechanics and patient-derived stem cells to predict what type of inherited heart defect a child will develop. Findings are published the journal Cell.
Scientists have identified a potential marker of disease activity for eosinophilic esophagitis (EoE), a severe and often painful food allergic disease. The marker could spare children with EoE the discomfort and risk of endoscopic procedures to assess whether their disease is active.
Findings are published in the Journal of Allergy and Clinical Immunology. The study was led by researchers at Cincinnati Children’s Center for Eosinophilic Disorders (CCED), including Patricia Fulkerson, MD, PhD.
Based on laboratory tests on human cells and mouse models, researchers report an experimental therapy that stops aggressive, treatment-resistant brain cancers called glioblastoma and high-grade gliomas. A multi-institutional team led by researchers at Cincinnati Children’s published their results in Cancer Cell on May 9th.
“We find that elimination of dividing Olig2-expressing cells blocks initiation and progression of glioma in animal models and further show that Olig2 is the molecular arbiter of genetic adaptability that makes high-grade gliomas aggressive and treatment resistant,” says Lead investigator Qing Richard Lu, PhD, the scientific director of Cincinnati Children’s Brain Tumor Center.
“Challenges with patient recruitment for clinical trials are a major barrier to timely and efficient translational research,” said Yizhao Ni, PhD, lead author and a researcher in Cincinnati Children’s Division of Biomedical Informatics. As reported in the Journal of the American Informatics Association, Yizhao and his colleagues are teaching computers to figure out why people accept or decline invitations to participate in clinical trials.
Results published in Science Signaling report two Cincinnati Children’s laboratories are developing a pharmacologic compound to treat life-threatening lung damage and breathing problems in people
with severe infections like pneumonia, those undergoing certain cancer
treatments and premature infants with underdeveloped, injury prone
lungs. Researchers led by Vladimir Kalinichenko, MD, PhD, and Tanya Kalin, MD, PhD, are following up their current study with further development of the targeted small molecule they recently discovered.
“Infection, a consequence of immune suppression, is the leading cause of death for people with spinal cord injuries,” said Yutaka Yoshida, PhD, co-lead author of a study published in Nature Neuroscience and a scientist in Cincinnati Children’s Division of Developmental Biology. The team of scientists from various institutions have identified an underlying cause of this dangerous immune suppression in people with high level spinal cord injuries and they propose a possible treatment.
A study published April 14 in Cell Stem Cell reports that mutations accumulate in human mitochondrial DNA with age. The finding has implications for potential therapies using induced pluripotent stem (iPS) cells. “People tend to look just at the nuclear genome,” says co-senior author Taosheng Huang, MD, PhD. “But if you want to use iPS cells in a human, you must check for mutations in the mitochondrial genome.” Huang directs Cincinnati Children’s Mitochondrial Disorders Program.
Researchers at Cincinnati Children’s led by Marc E Rothenberg, MD, PhD, have identified a protein that may be the cause of tissue damage in patients with eosinophilic esophagitis (EoE). Researchers note that the findings suggest a valuable drug target for further research.
Findings reported on April 1, 2016 at the Endocrine Society meeting in Boston suggest that pediatricians measure BMI during infant well-child visits. “BMI at 6, 12 or 18 months of age above the 85th percentile on the growth chart can accurately predict children at risk for early childhood obesity,” says Allison Smego, MD, a fellow in Cincinnati Children’s Division of Endocrinology and the study’s lead author. “These children have a high lifetime risk for persistent obesity and metabolic disease and should be monitored closely at a very young age," she said.
A novel mouse model of a highly lethal form of acute myeloid leukemia (AML) has been developed at Cincinnati Children’s and offers scientists an important tool to better understand this disease and research new therapeutic targets. “This model is rapid, fully penetrant, and completely spontaneous. We hope that it will open the way for other researchers to join us in attacking this particularly lethal AML subtype,” says H. Leighton Grimes, PhD, senior author and professor in the Division of Immunobiology. Results are reported in Cancer Discovery.
Research published online in Nature Neuroscience suggests the possibility of treating a group of genetic birth defects (CHARGE) with molecular therapy that would regenerate malformed nerve insulation in the central nervous system. “Our findings could provide a molecular framework for identifying signaling pathways and molecules as therapeutic targets to promote myelin regeneration in patients with CHARGE and other demyelinating diseases,” says senior author Richard Lu, PhD, scientific director of the Brain Tumor Center in the Division of Experimental Hematology and Cancer Biology.
NIH’s Bench to Bassinet Program has selected Cincinnati Children’s to lead a $32.5 million, five-year study to determine why children are born with heart problems and find effective treatments.
“As the coordinating center, we can make sure that research infrastructure and data are shared and integrated to accelerate discovery of the genetic and biological mechanisms of congenital heart defects,” said Eileen C. King, PhD, principal investigator of the project and associate professor in the Division of Biostatistics and Epidemiology. “This will ultimately lead to improvements in treating or even preventing these often devastating defects.”
A new study supports evidence that the beneficial effects of fish consumption during pregnancy outweigh the detrimental effects of low-level exposure to mercury. According to senior author Kim Yolton, PhD, the study “reflects the benefits of polyunsaturated fatty acid intake that also comes from fish and has been shown to benefit attention, memory, and other areas of development in children.” The study is published online in Neurotoxicology and Teratology.
A Cincinnati Children’s study published in Breastfeeding Medicine finds that women with diabetes during pregnancy face a significantly increased risk of having low milk supply. “This study shows the importance of further research to determine how maternal glucose intolerance may impede lactation, so that targeted therapies may be developed to increase milk supply,” says Sarah Riddle, MD, the study’s lead author and a pediatrician in the Center for Breastfeeding Medicine.
Sing Sing Way, MD, PhD, the
Pauline and Lawson Reed Chair in the Division of Infectious
Diseases, has won the 2016 E. Mead Johnson Award for Pediatric Research for
2016, considered among the most prestigious honors in pediatric research. In a joint letter nominating Dr. Way for the award, Margaret Hostetter, MD, and Lou Muglia, MD, PhD, called Dr. Way’s work “paradigm shifting and transformative.”
Dr. Way focuses on prenatal infection and maternal-fetal immunology. His
laboratory has made seminal contributions on the molecular-cellular mechanisms
responsible for maternal-fetal immunological tolerance, fetal injury after
prenatal infection, and infection susceptibility during pregnancy and in newborn
In a study published online in Autism Research, researchers from the Division of Biostatistics and Epidemiology show they can use electronic medical records and birth information to verify and strengthen an already suspected link between autistic children and pregnant mothers with obesity and diabetes. According to study data, pregnant mothers with obesity or gestational diabetes were 1.5 times more likely to have a child with an autism spectrum disorder (ASD) compared to mothers of children without developmental disorders. The increased risk of ASD for pregnant mothers with both obesity and gestational diabetes was two-fold.
“Without placing any burden on study participants or the costs of developing an epidemiologic study from scratch, we can use the vast amounts of data already collected for clinical purposes to conduct broad population-based studies on this link to autism. We are very excited about the future studies we can do with this ability,” says Katherine Bowers, PhD, MPH, the study’s senior author.
Cincinnati Children’s researchers find that exposure to high levels of small particle air pollution is associated with an increased risk of preterm birth, according to a new study published online in the journal Environmental Health. “We estimate that decreasing the amount of particulate matter in the air below the EPA’s standard threshold could decrease preterm birth in women exposed to high levels of small particulates by about 17 percent, which corresponds to a 2.22 percent decrease in the preterm birth rate in the population as a whole,” says physician-researcher Emily DeFranco, DO, with the Center for the Prevention of Preterm Birth.
Preterm birth rates were higher among mothers exposed to high levels of airborne particle pollution above the EPA standard, as well as among mothers 40 or older, black mothers, and women with no prenatal care or with lower education level.
Published in Stem Cell Reports, Cincinnati
Children’s researchers report identifying a molecular target and experimental
treatment strategy for DNA repair defects behind Fanconi anemia. The complex
genetic disorder is responsible for birth anomalies, organ damage, anemia and
cancer. The findings give hope for further discovery. Susanne
Wells, PhD, lead investigator and director of the Epithelial Carcinogenesis
and Stem Cell Program, says, “This study provides an experimental platform to
test new therapies that could prevent pre- and post-natal Fanconi anemia
conditions, which have no cure and limited treatment options.”
Researchers with Cincinnati Children’s Center
for Prevention of Preterm Birth have released a study in the online edition
of the American Journal of Obstetrics and Gynecology reporting that
black women are nearly four times more likely than white women to have a baby
born between 16 and 22 weeks gestation, a period in which life outside the womb
is not viable. “Our findings suggest that public health efforts should focus on
access to prenatal care, optimizing opportunities for preterm birth screening
and preventive efforts in high-risk black women to close the racial disparity
gap in infant mortality,” says Emily DeFranco, DO, a physician-researcher with
"Our study finds that transitions from hospital to
home affect the lives of families in ways that may impact patient outcomes at
discharge,” says Andrew
Beck, MD, MPH, a physician in the divisions of General
and Community Pediatrics and Hospital
Medicine and co-author on a new study published online in Nov. 30 edition
of Pediatrics. As part of the multi-stage research project
Hospital-to-Home Outcomes (H20), which is led by Cincinnati Children’s, this
study focused on obtaining detailed input from caregivers that will help
develop “family centered” solutions in future phases of the project.
A new study presented at a national
meeting on October 4, 2015 reveals that patients with congenital heart disease
and ADHD can safely benefit from stimulant medications. “Children with
congenital heart disease are at high risk for ADHD, but fears about
cardiovascular side effects, including sudden death, limit the use of stimulant
medications,” says Julia Anixt, MD, senior author of the study. “This study
indicates that stimulants are both effective and safe when prescribed with
appropriate monitoring and in collaboration with the patient’s
A study led by researchers in the Division
of Allergy and Immunology. reports findings that offer insight into new
therapeutic strategies and diagnostics for severe food allergies triggered by
immunoglobin E (IgE). The study, published in the journal Immunity,
reveals the discovery of IL-9-producing mucosal mast (MM9) cells that produce
large amounts of an inflammatory protein, interleukin 9 (IL-9), known to amplify
anaphylactic shock in response to certain foods. “Our study suggests that
although you need to have some level of IgE to trigger a food allergy response,
you also have to produce MMC9 cells to get a severe response and anaphylaxis,”
says Yui-Hsi Wang, PhD, the study’s co-first author. “Without
these cells you will not get severe food allergies.”
We are excited to announce the launch of a new,
innovative, Master’s Program at Cincinnati Children’s Hospital Medical Center,
in conjunction with the University of Cincinnati. The Biomedical
Research Master’s Program is born from the recognition that there is a
growing job market, in both academia and industry, for scientists who have a
strong foundation in the basic methods of biomedical laboratory research, and
are trained in emerging techniques and technologies. We have designed this new
program to provide students with extensive hands-on training in basic molecular
biology, cell biology and biochemistry as well as rotations in our state-of-the
art core facilities where they will be provided one-on-one training in the use
of stem cell techniques, the generation of transgenic animals, flow-sorting,
histology, high-end imaging techniques, animal handling, and single-cell
techniques. Our first class of five students started classes on August 24 and
we are expecting that they will graduate in May of 2017. If you would like
more information this new and exciting program, please contact us at BMRMSProg@cchmc.org.
Hospital Medical Center is among a group of medical research institutions
awarded $52.4 million by the National Institutes of Health (NIH) over the next
four years to identify the potential medical effects of rare genomic variants
in about 100 clinically relevant genes.
Led by principal investigator
Harley, MD, PhD, director of the Center for Autoimmune
Genomics & Etiology at Cincinnati Children’s, the medical center will
receive $3.4 million over four years to evaluate the role of 100 genes in the
genomes of 2,500 patients who agree to receive their test results.
A study published in the journal
Magnetic Resonance in Medicine and led by scientists at Cincinnati
Children’s has demonstrated the feasibility of using ultrasound as a
non-invasive therapy to destroy fat. This development may lead to new
treatments for metabolic syndrome. The lead author, Charles
Dumoulin, PhD, is the director of the Imaging
Research Center at Cincinnati Children’s.
The Center for Clinical and Translation
Science and Training (CCTST) has been awarded $16.7 million from the
National Institutes of Health (NIH) to further enhance its efforts in
translating basic scientific discoveries from the laboratory bench to patients’
bedsides. "This new funding will enable us to build on the transformation
accomplishments we’ve been able to instigate or serve as a catalyst for at the
CCTST,” says James
Heubi, MD, co-director of the CCTST. The Center is a collaborative research
resource among Cincinnati Children’s Hospital Medical Center, the University of
Cincinnati and the Cincinnati Department of Veterans Affairs Medical
A study published online Aug. 24 in the journal Pediatrics finds a
significant decrease in the use of computed tomography (CT) scans at children’s
hospitals for 10 common childhood diagnoses. Study authors hypothesize the
decline in CT usage may be attributable to a growing body of evidence linking
ionizing radiation from CT scans to an increased risk of cancer in patients.
“This study reinforces the pediatric community’s commitment to think about both
immediate and long term risks and benefits of our treatment,” said Michelle Parker, MD, the study’s lead
investigator and a physician in the Division of Hospital
Medicine at Cincinnati Children’s.
Data reported Aug. 6 in Molecular Cell
highlight a technology developed at Cincinnati Children’s that might offer
scientists new ways of approaching previously unanswerable questions in the
field of tissue development and disease research. Senior author Raphael Kopan, PhD, director of Developmental Biology,
calls the SpDamID method “transformative for our research.”
“Providers must be more systematic in the
screening, diagnosis and management of mental health conditions in children and
teens with Down syndrome,” concludes Julia
S. Anixt, MD, lead author of a study recently completed at Cincinnati
Children’s. Researchers studied trends related to the prescription of
stimulants, selective serotonin reuptake inhibitors (SSRIs) and atypical
The Pew Charitable Trusts have named 22 promising
early-career researchers as Pew scholars in the biomedical sciences. Among the
recipients are two Cincinnati Children's researchers: Theresa Alenghat, VMD, PhD of the Division
of Immunobiology and Douglas
Millay, PhD of the Division of Molecular Cardiovascular
Biology. Awardees were nominated based on their dedication to pursuing
high-risk, high-reward research that can ultimately lead to extraordinary
findings in bioscience.
researchers, including Theresa
Guilbert, MD, MS, report in the Nov. 17 edition of
JAMA that acute episodes of severe lower respiratory tract illness (LRTI)
are common among preschoolers, and 14 percent to 26 percent of preschoolers
experience recurrent wheezing during the first 6 years of life. However, study
findings suggest that early use of an antibiotic significantly reduces the risk
of severe LRTI in young children with a history of recurrent episodes of the
A study led by Andreas Loepke, MD, PhD and published in the journal
Pediatrics found that children under age 4 who received general
anesthesia for surgery risk diminished language comprehension, lower IQ and
decreased gray matter density in posterior regions of the brain.
Cincinnati Children’s researchers recently identified that parent-reported
responses to the Pediatric Eosinophilic Esophagitis Symptom Score (PEESS® v2.0)
questionnaire correspond to clinical and biologic features of eosinophilic
esophagitis (EoE). The study’s first author, Lisa
Martin, PhD, reports, “Because eosinophilic esophagitis is a disease with
multiple symptoms, the ability to capture patient and parent perceptions of
these symptoms is a major unmet need.”
Published in Environmental Health, a recent study
found a link between exposure to pyrethroid pesticides and ADHD in children and
young teens. Corresponding author Tanya Froehlich, MD states, “Given the growing use of
pyrethroid pesticides and the perception that they may represent a safe
alternative, our findings may be of considerable public health
A study published in Science Signaling by Kakajan
Komurov, PhD suggests that maxing out the inherently stressed nature of
treatment-resistant breast cancer cells thwarts their adaptive ability to
evolve genetic workarounds to treatment.
A study recently published in Pediatrics finds
that pharmacies in neighborhoods with high rates of asthma-related
emergency-room use and hospitalization filled fewer asthma controller
medications compared to asthma rescue medications. Lead author, Andrew Beck, MD, MPH states, “Tracking medication fills could
highlight ways in which pharmacies could deliver proactive, as opposed to
reactive, asthma care.”
A Cincinnati Children’s-led study concludes
that breastfeeding Arab mother and infants have a very high prevalence of
Vitamin D deficiency. The study’s lead author, Adekunle Dawodu, MBBS, points to Arab women’s traditional
style of dress as a major factor, as it largely prevents exposure of the skin
Why do some asthma patients respond well to
corticosteroids while others do not? A new study reveals that VNN-1 gene expression is required
for corticosteroids to be effective during an asthma attack. Gurjit
Khurana Hershey, MD, PhD and her research team discovered the revealing
bio-marker and are using their findings to develop new treatments for
hard-to-treat asthma cases.
Promising research focused on
redirecting an expectant mother’s immune cells away from the fetus suggests new
therapeutic strategies for preventing pregnancy complications, including
prematurity and stillbirth. Senior author Sing Sing Way, MD, PhD hopes these findings – published in
the Journal of Clinical Investigation – will spark a renewed interest in
the biomedical community to establish the underlying causes of certain
pregnancy complications and the development of new therapeutic
Cardiovascular risks for severely
overweight teens are much higher than previously realized, according to an article published online in JAMA Pediatrics. A
part of the ongoing Teen Longitudinal Assessment of Bariatric Surgery
(Teen-LABS), the findings include increased risk of elevated blood pressure,
unhealthy cholesterol and insulin resistance in severely overweight teenagers.
Thomas Inge, MD, PhD, acts as Chair for the Teen-LABS
A new study published in the American Journal of
Preventive Medicine finds child care centers play a pivotal role when it
comes to the physical activity levels of preschoolers. The Preschool Eating and
Activity Study (PEAS) found that children in child care centers with at least
60 minutes of outdoor time were more active over 24 hours than children that
did not get this time. “These [outdoor play] opportunities may be especially
important for children who lack opportunities to be active at home,” says Kristen
Copeland, MD, a researcher in the Division of
General and Community Pediatrics and senior author of the study.
In a study presented Nov. 10 at the annual American
Heart Association meeting in Orlando, researchers found that eight years after
having gastric bypass surgery as teenagers, patients continue to have
significant weight loss and improvement in their lipid profiles. “Those who did
not undergo surgery experienced weight gain and no improvement in
co-morbidities over time,” says Elaine Urbina, MD, presenter and Director of
Preventative Cardiology at Cincinnati Children’s. “These findings highlight the
concerning long-term health trajectory for those with severe pediatric obesity
and suggest that bariatric surgery can meaningfully and durably improve
long-term outcomes in teens with this disease.”
An international group of researchers, including
Cincinnati Children’s cardiologist Nicolas Madsen, MD, analyzed over 14,000
patient records, finding that adults up to age 70 who were born with heart
disease show a dramatically increased risk of heart attack. “We’re not used to
thinking about heart attack risk in a 30 or 40 year old, but we should be in
this population,” says Dr. Madsen, who presented the study at Nov. 9 at the
annual meeting of the American Heart Association in Orlando.
A study published Nov. 6 in The New England
Journal of Medicine offers hopeful news for bariatric adolescent patients
and their families. Thomas Inge, MD, PhD, lead author of the study, reports
results showing that three years post-surgery, “…almost 90 percent [of
patients] experienced clinically meaningful weight loss, and participants were
in better health, with improved quality of life scores.” Dr. Inge is the
surgical director of the Surgical Weight Loss Program for
Teens at Cincinnati Children’s.
When the FDA approved the drug mepolizumab on November 4th to help
treat severe asthma, it was a blip on the radar screen of health industry news.
The medication targets a subset of asthma patients (ages 12 and up) whose
current drug regimens are insufficient to control their condition.
from official press announcements about mepolizumab’s approval on Nov. 4 are
the many years of research and testing by countless physicians and scientists
at institutions around the world. Cincinnati Children’s and its Division of
Allergy and Immunology were key parts to this effort.
Doctors may soon have a new tool to help them time births
in the case of delicate, pre-mature babies. A study led by researchers at Cincinnati Children’s Perinatal
Institute have identified a way to test RNA and specific genetic signatures
in amniotic fluid to see whether fetal lungs – and potentially other organs –
are mature enough for a safe and viable delivery. “This will allow pediatricians
and neonatologists to prepare for the various neonatal morbidities these
preterm infants may face, and allow obstetricians to better weigh risks to the
baby when making decisions about preterm delivery,” says Beena Kamath-Rayne, MD,
MPH, one of the study’s lead authors. The findings are published in BMC
A new computer program developed at Cincinnati
Children’s analyzes functional brain MRIs to predict whether hearing impaired
children will develop effective language skills within two years of cochlear
implantation surgery. “This study identifies two features from our computer
analysis that are potential biomarkers for predicting cochlear implant
outcomes,” says collaborator Long
(Jason) Lu, PhD from the Division
of Biomedical Informatics. The findings are published in the Oct. 12 online
Findings published online in the December 15
issue of JAMA Pediatrics report on a multi-dimensional study, co-led by Cincinnati Children’s. The study found that osteomyelitis treatment can be equally as effective through IV and oral antibiotics. According to study co-author Samir Shah, MD, MSCE, serious complications can be avoided by opting for oral antibiotics over IV treatment.
A new test that measures five key biomarkers can quickly and accurately predict the risk of death in children with septic shock, according to a study published online Jan. 29 in PLOS ONE. The multi-institutional study was led by Hector Wong, MD, Director, Division of Critical Care Medicine at Cincinnati Children’s and Christopher Lindsell, PhD, UC College of Medicine.
New research links ADHD and conduct disorder in young adolescents with increased alcohol and tobacco use. William Brinkman, MD, was lead author in the study published in the journal Drug and Alcohol Dependence.
A sickle Cell Anemia Clinical Trial ended early as conclusive data shows that hydroxyurea therapy offers safe and effective disease management of sickle cell anemia (SCA) and reduces the risk of stroke. Russell E. Ware, MD, PhD, was principal investigator of the study.
A new type of cell transplantation developed to treat mice mimicking a rare lung disease could one day be used to treat this and other human lung diseases caused by dysfunctional immune cells. The study was published online Oct. 1 by Nature. Bruce Trapnell, MD is senior author.
New research published in Nature Genetics identifies a novel genetic and molecular pathway in the esophagus that causes eosinophilic esophagitis (EoE). Marc E. Rothenberg, MD, was senior investigator.
Treating infants with high doses of steroids fails to improve medical outcomes in the end-stage pediatric liver disease, according to a study published in the May 7, Journal of the American Medical Association. Jorge Bezerra, MD was principal investigator.
By studying the genomes of twin 3-year-old sisters, researchers have uncovered a molecular pathway involving the gene SETD2 that could lead to better treatments for aggressive leukemia. The study was published online Feb. 9 in Nature Genetics. Gang Huang, PhD, a researcher in the divisions of Pathology and Experimental Hematology and Cancer Biology was a co-corresponding author.
In a mouse study, researchers have successfully used blood platelets and bone marrow cells to deliver potentially curative gene therapy to treat Hurler syndrome. The study appeared online Feb. 3 in Proceedings of the National Academy of Sciences (PNAS). Dao Pan, PhD, a researcher in the Division of Experimental Hematology and Cancer Biology, was corresponding author.
A new study published online Feb. 2 in Pediatrics finds no evidence that the human papillomavirus (HPV) vaccine leads to unsafe sexual behaviors among teenage girls and young women. Jessica Kahn, MD, a physician in the Division of Adolescent and Transition Medicine was senior author.
A basic signaling
pathway known to play important roles in normal cell and cancer cell formation
also plays an unexpected role as a molecular switch that controls the aging
process of hematopoietic stem cells (HSCs), according to a study published online Oct. 20, 2013, in Nature. The study was led by Hartmut
Geiger, PhD, Experimental Hematology and Cancer Biology.
In a study
published May 21, 2013 in Environmental Health Perspectives, a team led
Newman, DO, MS, director of the Pediatric Environmental Health and Lead Clinic at Cincinnati Children’s, reported that
children exposed to high levels of traffic-related air pollution (TRAP) during
their first year of life were more likely to have “at risk” scores for
hyperactivity by age 7.
The same modern
medical devices that have helped extend and enhance life for so many children
also can cause complications that have not been well-understood, according to a
study led by Patrick
Brady, MD, MSc, a physician in the Division
of Hospital Medicine. Findings were posted
online June 7, 2013, in the Journal of Hospital Medicine.
known effects that surgical anesthesia can have on the developing brains of
young children, new
findings posted June 5 the Annals of Neurology suggest the threat may
also apply to adult brains. The study was led by Andreas
Loepke, MD, PhD, a physician and researcher in the Department
An international team
of researchers led by Cincinnati Children’s has confirmed 14 more genes linked
to juvenile idiopathic arthritis (JIA), bringing the total to 17. The study
involved patient DNA samples from across the United States, Germany and United
Kingdom, says Susan
Thompson, PhD, a researcher in the Division
of Rheumatology who was a leader in the project. Findings were published
April 21, 2013, in
microbial imbalances in the digestive tract may serve as biomarkers to predict
the onset of necrotizing enterocolitis (NEC), according to a study led by Ardythe
Morrow, PhD. Findings
were published April 16, 2013, in the journal
Manipulating bile acid levels may be enough to recreate the key effects of
bariatric surgery without the need for an invasive procedure, according to a
study led by Rohit
Kohli, MBBS, MS, a member of the Division
of Gastroenterology. Findings
were published online April 16, 2013, in Endocrinology.
For children with X-SCID, a new version of gene therapy shows renewed promise. A boy treated when he was 8 months old is doing well several months after receiving experimental therapy through a clinical trial at Cincinnati Children’s.
at UC and Cincinnati Children’s report that a new molecular pathway may offer a
new way to kill leukemia cancer stem cells that survive traditional forms of
were published in June 2012 in the journal Blood. Our researchers also
are using new lines of “humanized” mice to explore the potential of microRNA
inhibitors and small-molecule inhibitors as anti-cancer weapons.
Hybrid Immunotherapy for HLH
(HIT-HLH) trial, led by Michael
Jordan, MD, is the first US-based clinical trial to focus on this condition.
Researchers at Cincinnati Children’s also are studying a promising antibody, a
possible gene replacement therapy and targeted drug therapies to improve HLH
Even in the
womb, the eye needs light to develop normally. This and other unexpected
findings could change our understanding of how the retina develops, according to
study published online Jan. 16, 2013, in Nature. The paper was
co-authored by Richard
Lang, PhD, a researcher in the Division
of Pediatric Ophthalmology at Cincinnati Children’s and David Copenhagen,
PhD, a scientist at UCSF.
Severely obese teens
are four times more likely to have swollen legs with skin ulcers as adults and
three times more likely to develop severe walking limitations and abnormal
kidney function, according to a study
published online Nov. 18, 2013, in Pediatrics by Thomas Inge,
MD, PhD, and colleagues.
people have had kidneys removed unnecessarily because doctors misdiagnose
patients who actually have tuberous sclerosis complex (TSC), according to a
study published online Jan. 11, 2013, in Lancet. Proper diagnosis
could have led to a medication that would have made surgery or kidney removal
unnecessary, according to John Bissler, MD, a nephrologist at Cincinnati
Children’s and lead author of the study.
percent of teen girls report having offline meetings with strangers and
semi-strangers they meet on the Internet, according to a
study published online in Pediatrics. The study, led by says Jennie
Noll, PhD, a psychologist in Behavioral
Medicine and Clinical Psychology at Cincinnati Children’s, shows the risk is
further heightened for teen girls who have been victims of abuse or neglect.
Treatment with antioxidants may help reduce behavioral
issues linked to neurofibromatosis 1 (NF1) and an associated condition, Costello
syndrome. Findings were posted Sept. 12 in Cell Reports. The study was
led by Nancy
Ratner, PhD, Division of Experimental Hematology and Cancer Biology.
protein RhoA could help fight a variety of blood and immune system disorders,
according to a study published online Oct. 7 in the Journal of Experimental
Zheng, PhD, director of Experimental
Hematology and Cancer Biology, was principal investigator.
study involving mouse models published Aug. 2, 2013, in the Journal of
Biological Chemistry reports that depleting the FoxM1 protein in prostate
epithelial cells inhibits tumor cell proliferation and metastasis. Tanya Kalin, MD,
PhD, a physician-scientist in Pulmonary
Biology was senior author.
posted online April 26, 2013, in Leukemia report that an experimental
combination treatment was effective against T-cell acute lymphoblastic leukemia
(T-ALL) in mouse models and human cells in the lab. Fukun Guo, PhD, a
researcher in Experimental
Hematology and Cancer Biology, is first author.
reveals in detail how insulin resistance can lead to insufficient breast milk
study, led by Laurie
Nommsen-Rivers, PhD, at Cincinnati Children’s and scientists at the
University of California Davis, was published in July 5, 2013, in PLOS
ONE. The findings suggest a potential biomarker to predict which women may
have difficulty breastfeeding.
dramatically reduce seizures in patients with tuberous sclerosis complex (TSC),
according to a study
posted online June 24, 2013, in Annals of Neurology. The study was led by
Krueger, MD, PhD, at Cincinnati Children’s in collaboration with a team at
Texas Children’s Hospital.
at Cincinnati Children’s have successfully targeted a malfunctioning immune
system enzyme to kill diseased cells from patients with myelodysplastic syndrome
study, led by Daniel
Starczynowski, PhD was published July 8, 2013, in Cancer Cell.
study in mice successfully used targeted molecular therapy to block mostly
untreatable nerve tumors caused by the genetic disorder Neurofibromatosis 1
(NF1). The study was led by Nancy
Ratner, PhD, program leader for the Cancer Biology and Neural Tumors Program
in the Cancer
and Blood Disorders Institute. Findings were published online Dec. 10, 2012,
in the Journal of Clinical Investigation.
Persistent and loud
snoring in young children can make preschool behavior problems worse, according
Beebe, PhD, director of the neuropsychology program at Cincinnati
Children’s. Behaviors affected by snoring include hyperactivity, depression and
were published online Aug. 13 in Pediatrics.
The HPV vaccine
does more than protect the vaccinated. A study led by Jessica Kahn, MD,
MPH, a physician in the division of Adolescent
Medicine at Cincinnati Children’s, is the first to document that the vaccine
also provides herd immunity. Findings
were published online July 9 in Pediatrics.
modified echocardiogram is more effective than EKG at detecting athletes at risk
of sudden cardiac death, according to a study led by Michelle Grenier, MD, a
physician at the Cincinnati
Children’s Heart Institute Findings were presented in July at the annual
meeting of the American Society of
who smoke accumulate less bone during a critical growth period and carry a
higher risk of developing osteoporosis later in life, according to research
published Dec. 4, 2012, in the Journal of Adolescent Health. The study
was led by Lorah Dorn, PhD, director of research in the Division
of Adolescent Medicine.
A research team
led by Cincinnati Children’s has discovered a genetic mutation responsible for
deafness associated with Usher syndrome type 1. These findings offer a potential
target for new therapies, says Zubair Ahmed, PhD, senior investigator. The study
results were published online Sept. 30 in Nature Genetics.
consumption may increase risks of preeclampsia and other pregnancy
complications, according to SK
Dey,PhD, director, Division
of Reproductive Sciences. Analysis of mouse models indicates that THC can
affect placenta development. Findings
appeared Sept. 14 in The Journal of Biological Chemistry.
Warning labels are not
working. Tougher rider training and helmet laws are needed to reduce deaths
among underaged All-Terrain Vehicle (ATV) riders, according to Rebeccah Brown,
MD. She presented new
findings about ATV crashes Oct. 22 at American Academy of Pediatrics annual
meeting in New Orleans.
disruptions in granule cells – located in the dentate gyrus region of the brain
– caused brain seizures in mice similar to those seen in human temporal lobe
epilepsy, according to a study led by Steven
Danzer, PhD, a neuroscientist in the Department
of Anesthesia. The findings
appeared Sept. 19 in Neuron.
small-molecule-inhibiting drug dubbed “Rhosin” stopped breast cancer cells from
metastasizing and promoted nerve cell growth in early laboratory cell tests.
Although years away from market, Rhosin eventually could treat a variety of
cancers and could promote spinal cord regeneration, says lead investigator Yi Zheng, PhD,
director of Experimental
Hematology and Cancer Biology. Findings appeared June 21 in
social risk index based on income, home values and the parents’ education levels
can help hospitals predict which children with asthma are most likely to need
readmission, according to research led by Andrew
Beck, MD. Study results
were posted online Oct. 18 in the American Journal of Public
involved in preventing a pregnant woman’s body from rejecting her baby as a
foreign object eventually may lead to a new class of vaccines that could prevent
preterm births and possibly other autoimmune diseases, according to Sing Sing
Way, MD, PhD, a physician researcher in Infectious
were published online Sept. 26 in the journal Nature.
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