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Center for Stem Cell & Organoid Medicine (CuSTOM)
Center for Stem Cell & Organoid Medicine (CuSTOM)

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Center for Stem Cell & Organoid Medicine (CuSTOM)

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CuSTOM Faculty

At CuSTOM we have assembled a highly collaborative multi-disciplinary team of developmental biologists, stem cell experts, geneticists, clinicians, surgeons and entrepreneurs with complementary expertise to accelerate discovery and translation.

Theresa Alenghat, VMD, PhD

Division of Immunobiology

The Alenghat Lab investigates epithelial and immune cell homeostasis in the context of intestinal health and disease.

Akihiro Asai, MD, PhD

Division of Gastroenterology, Hepatology and Nutrition

The goal of the Asai lab is to develop new treatment for pediatric liver disease. Using liver organoids from patient-derived pluripotent stem cells the Asai team is investigating the pathophysiology of disrupted bile acid metabolism and identifying new therapeutic targets of pediatric cholestatic liver disorders.

Riccardo Barrile, PhD

University of Cincinnati College of Engineering and Applied Science, Department of Biomedical Engineering

The Barrile Lab focus is on combining patient-derived stem cells and reverse engineering approaches to establish an highly translational platform designed to capture the complex and dynamic nature of brain-ischemic stroke and metastatic lung-cancer. The lab aims to explore the cellular and molecular mechanisms that govern organ-functions during the homeostasis and disease state, and how these processes can be therapeutically targeted in order to create personalized therapies.

Samantha Brugmann, PhD

Division of Developmental Biology

The Brugmann Lab studies craniofacial development and disease. We are particularly interested in combining stem cell based technologies together with biologically inspired principles to engineer human craniofacial tissues suitable for use in the surgical repair of craniofacial anomalies.

Kenneth Campbell, PhD

Division of Developmental Biology

The Campbell Lab works to uncover the molecular genetic mechanisms that control the normal formation of basal ganglia circuits.

Jose Cancelas Perez, MD, PhD

Division of Experimental Hematology and Cancer Biology

Dr. Jose Cancelas collaborates closely with Dr. Carolyn Lutzko in disease modeling and ex vivo generation of blood cells from pluripotent stem cells. View recent examples of this work.

Lee Denson, MD

Division of Gastroenterology, Hepatology & Nutrition

The long-term goal of Dr. Denson’s research program is to define genomic and microbial mechanisms driving disease complications in Crohn’s Disease (CD). To this end the lab has developed a novel human intestinal organoid (HIO) model system to test genetic mechanisms and microbial metabolites regulating tissue collagen production. These studies will advance precision medicine, by developing a platform to screen small molecules in patient-derived organoids stratified by clinically important genotypes, and ultimately prevent fibrotic end-organ injury.

Leyla Esfandiari, PhD

University of Cincinnati College of Engineering and Applied Science, Electrical Engineering and Biomedical Engineering

The Esfandiari Lab uses integrated bioengineering and stem cell technologies to design and fabricate nano-scale biosensors and actuators to monitor the biophysical and biochemical microenvironment of living organoids. These tools accelerate the next generation of organoid based-therapeutic screens and enable personalize medicine.

Brian Gebelein, PhD

Division of Developmental Biology

The Gebelein Lab areas include developmental biology, gene expression regulation, neural cell-fate specification, mechanisms patterning the early embryo and the mechanisms underlying Notch signaling, with a specific emphasis on modeling the disease alleles of Adams-Oliver syndrome.

Mingxia Gu, MD, PhD, FAHA

Divisions of Pulmonary Biology, Molecular Cardiovascular Biology, and Developmental Biology

The overarching goal of the Gu Lab is to develop novel therapy for the regeneration of heart, lung, and vasculature in patients with congenital cardiac and pulmonary defects. Towards this goal, the Gu Lab conducts translational and interdisciplinary research using patient-specific induced pluripotent stem cells (iPSCs), vessel and lung organoids, animal models, and single cell RNA/ATAC sequencing for disease modeling and high-throughput drug screening.

Ziyuan Guo, PhD

Division of Developmental Biology

The goal of Guo Lab research is to apply cutting-edge stem cell technologies (e.g. iPSCs, organoid and transdifferentiation) to study the etiology of neurodevelopmental and neurodegenerative disorders in a human genetic context. And new insights from these investigations may eventually help identifying novel targets for therapeutic development.

Michael A. Helmrath, MD

Director of CuSTOM Clinical Translation, Division of General and Thoracic Surgery

The Helmrath Lab interfaces with the clinical care of patients with intestinal diseases. As a translational research lab, the Helmrath team continues to develop in vitro and in vivo organoid models to study the development and function of human intestine.

Michael A. Helmrath, MD, MS Bio >

Christian Hong, PhD

Department of Pharmacology and System Physiology, University of Cincinnati

The Hong lab investigates molecular mechanisms of circadian rhythms and their functions in other cellular processes such as cell cycle and metabolism. Currently, we focus on identifying molecular components that connect the circadian clock and cell cycle, and the consequences of this coupling in intestinal stem cell regeneration and proliferation using mouse and human organoids.

Yueh-Chiang Hu, PhD

Division of Developmental Biology

Yueh-Chiang Hu, PhD, leads a highly productive, research-oriented Transgenic Animal and Genome Editing (TAGE) core that utilizes state-of-the-art genome editing and transgenic technologies to generate and preserve animal and cell models for investigators at Cincinnati Children's and across the nation. He is also studying the mechanism by which the gonads drive germ cell differentiation at the critical time of the development using genetic and genomic tools, including inducible CRISPRi and CRISPRa mice generated by his lab.

Vivian Hwa, PhD

Basic Research Director, Cincinnati Center for Growth Disorders

The human liver is a key responder to many endocrine hormonal cues critically important for normal developmental growth and metabolic homeostasis. The Hwa Lab, Cincinnati Center for Growth Disorders, is interested in modeling endocrine growth disorders utilizing patient-based iPSC-derived liver organoids for investigation and personalized therapy testing.

Makiko Iwafuchi, PhD

Division of Developmental Biology

The goal of Iwafuchi lab is to reveal chromatin regulatory principles underlying dramatic cell fate changing events that occur in development, tissue repair, and diseases, and ultimately to utilize this knowledge to precisely engineer cell fates. We focus on how different families of pioneer transcription factors regulate chromatin structure, taking advantage of genetically modified human stem cells and organoid systems.

Theodosia A. Kalfa, MD, PhD

Division of Hematology

The Kalfa lab focuses on erythropoiesis and red cell biology. Congenital dyserythropoietic anemias (CDAs) are diseases of erythropoiesis caused by genetic defects affecting erythroblast cytokinesis. We have developed the CDA Registry and use iPSCs generated from patient-donated blood to model these erythropoiesis disorders in vitro and study the mechanisms of normal and disordered erythropoiesis.

Vladimir Kalinichenko, MD, PhD

Division of Pulmonary Biology

The Kalinichenko Lab investigates cell signaling pathways and transcriptional mechanisms critical for the development of pulmonary vasculature and endothelial repair after lung injury. We use animal models, lung organoids and iPSC-derived endothelial progenitor cells to identify causes of vascular insufficiency and to develop new therapies for pediatric and adult pulmonary diseases.

Magdalena Kasendra, PhD

CuSTOM Director of Research and Development, Division of General and Thoracic Surgery

The mission of CuSTOM Accelerator is to create innovative organoids-based platforms enabling discovery and development of safer, more efficacious and precise drug treatments and transformative organoids-based therapies to advance human health and medical practices. At CuSTOM Accelerator we leverage our world-class preclinical and clinical expertise, experience in bioproduct development alongside with leading industry partners to deliver on the promise of organoid medicine.

Rafi Kopan, PhD

Division of Developmental Biology

The Kopan lab uses animal models and human kidney organoids to investigate the role of Notch signaling in nephron development and aging. The lab is currently developing novel CRISPR/Cas9 gene edited iPSCs that will enable the team to inducibly modulate gene expression during organoid development, dissect gene regulatory circuits, or mimic disease states.

Carolyn Lutzko, PhD

Experimental Hematology and Cancer Biology - Translational Labs

Dr. Lutzko is the Director of the Cell Manipulations Laboratory at Cincinnati Children’s and collaborates closely with Dr. Jose Cancelas Perez in disease modeling and ex vivo generation of blood cells from pluripotent stem cells. Her research interests include regulating human pluripotent stem cells, somatic cell reprogramming in iPSC, human embryonic stem cell physiology and differentiation, hESC, and cystic fibrosis.

Chris Mayhew, PhD

Director, Pluripotent Stem Cell Facility
Division of Developmental Biology

The Cincinnati Children's Pluripotent Stem Cell Facility facilitates and supports studies using human pluripotent stem cells. Services include routine access to quality-controlled hESC and iPSC lines, generation of patient-specific iPSCs, CRISPR-Cas9 mediated genome editing of iPSCs, and hands-on training modules in the maintenance of high-quality hESC/iPSCs and differentiation into GI organoids.

Kyle McCracken, MD, PhD

Division of Nephrology and Hypertension

The McCracken Lab uses stem cell and organoid models to investigate basic development and morphogenesis of the kidney, and we aim to generate increasingly innovative and sophisticated engineered kidney tissue models to apply to studies of renal function and pathophysiology.

Takahisa Nakamura, PhD

Department of Pediatrics

The Nakamura lab investigates mechanisms of immunometabolism to understand the pathogenesis of obesity-associated diseases, including type 2 diabetes, fatty liver diseases, and cancer, with a special focus on RNA-RNA binding protein networks.

Anne Perl, PhD

Neonatology and Pulmonary Biology

The Perl lab investigates the mechanisms of pulmonary development and disease using lung organoids and animal models. Current studies focus on the epithelial-mesenchymal interactions that promote alveolar maturation, the role of lung pulmonary fibroblast and using organoids derived from patient’s epithelial cells to model mechanisms of fibrotic lung disease.

Mattia Quattrocelli, PhD

Department of Pediatrics

The Quattrocelli Lab investigates molecular mechanisms and translatable biomarkers in dysfunction and rescue of striated muscles. The lab aims at combining genetics, epigenetics, and metabolism to garner a deeper understanding of physiology and pharmacology of muscle and heart. A current focus of the lab is the identification of unknown determinants of glucocorticoid receptor function.

Nathan Salomonis, PhD

Division of Biomedical Informatics

The Salomonis lab is committed to understanding the genetic programs that control pluripotency and differentiation at the single cell level. We develop integrative genomic and bioinformatics tools to study programmed and stochastic decisions that mediate cell state progression.

Dorothy M. Supp, PhD

Adjunct Research Professor, Department of Surgery, University of Cincinnati College of Medicine

The Supp lab focuses on wound healing and tissue engineering. We are studying the molecular mechanisms of keloid and hypertrophic scar formation so that improved approaches for suppressing scar and promoting regenerative healing can be developed. Additionally, we are developing tissue-engineered substitutes for skin, oral mucosa, and esophagus for treatment of traumatic wounds, such as burns, and the hereditary disease Recessive Dystrophic Epidermolysis Bullosa. These engineered tissues may be used for ex vivo gene therapy following genome editing of patient-derived epithelial stem cells, mesenchymal cells, and/or iPS cells.

Takanori Takebe, MD

CuSTOM Director of Commercial Innovation, Divisions of Gastroenterology, Hepatology and Nutrition & Developmental Biology

The Takebe lab pioneered the development of self organizing human liver bud organoids, which they use as a novel platform to study liver disease, screen for drugs and develop new therapies to intractable liver.

Jason Tchieu, PhD

Division of Developmental Biology

The Tchieu lab investigates the role of glial cells in development and disease using the directed differentiation of human pluripotent stem cells as a model. The goal of this research is to identify mechanisms involved in glial competency, uncover the diversity of glia in humans and the role of glial cells in neurodevelopmental disease.

Kelli VanDussen, PhD

The VanDussen lab studies the roles of intestinal epithelial cells in health, injury repair, and inflammatory disease. We use human tissue samples, mouse models, and tissue-derived primary intestinal epithelial “organoid” cultures from mice and patients. Our goal is to uncover intestinal disease biology and potential therapeutic strategies that could be harnessed to promote healing.

Alison Weiss, PhD

Department of Molecular Genetics, Biochemistry & Microbiology, University of Cincinnati

The Weiss lab is focused on understanding human intestinal pathogens, including E. coli O157:H7, the “hamburger E. coli”. Currently there is no treatment for this potentially fatal pediatric disease. Mice are not susceptible to O157:H7, and the use of stem-cell derived human intestinal organoids affords an unprecedented approach to study potential therapeutic interventions.

James Wells, PhD

CuSTOM Chief Scientific Officer, Divisions of Developmental Biology and Endocrinology

The research in the Wells lab aims to uncover the processes by which gastrointestinal and endocrine organs form in the developing embryo and to use this information to generate human tissue organoids from pluripotent stem cells. Gastrointestinal organoids are being used to study congenital defects of the digestive tract, endocrine control of metabolism, and for engineering functional tissues for transplantation.

Susanne Wells, PhD

Division of Oncology

The Susanne Wells laboratory studies the biology of human epidermis and mucosa. Keratinocytes within these compartments are tightly connected to form the organismal first line of defense against a hostile environment. These same cells are also most exposed to exogenous stressors including chemical carcinogens, radiation, or oncogenic viruses which promote transformation to squamous cell carcinoma (SCC). We seek to understand inherited and environmental factors which produce SCC susceptibility in children and adults, with the goal of developing innovative cancer prevention and treatment approaches. Patient-derived models include the directed differentiation of pluripotent stem cells into 3D epidermal and esophageal organoids for phenotype discovery in cancer susceptibility disorders.

Jeffrey Whitsett, MD

Co-Director, Perinatal Institute
Chief, Section of Neonatology, Perinatal and Pulmonary Biology

The overarching goal of the Whitsett lab is to understand the molecular mechanisms governing lung development, homeostasis and disease in order to improve treatment of lung disease. Current translational projects include gene editing of patient derived iPSCs to correct the mutations that cause child hood interstitial lung disease and “LungMap” a comprehensive single cell atlas of the fetal lung.

William J. Zacharias, M.D., Ph.D.

Division of Pulmonary Biology

The Zacharias lab investigates the mechanisms and cells which are involved in the regeneration of the lung following severe acute and chronic injury. The lab uses a combination of genetic animal modeling and mouse and human organoid cultures to define the mechanisms of lung regeneration and identify pathways with therapeutic potential. The overall goal of this work is to develop a clear understanding of the key steps in lung regeneration, and to identify and target pathways involved to promote lung healing in patients with lung disease and critical illness.

Aaron Zorn, PhD

CuSTOM Director, Division of Developmental Biology

The Zorn lab investigates the cell signaling programs that control embryonic development of the digestive and respiratory system using a combination of animal models, human pluripotent stem cell-derived organoids and cutting edge genomics. Current studies focus on elucidating the origins of trachea-esophageal birth defects.

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