Experimental Hematology and Cancer Biology

David A. Williams, MD

Title

Adjunct Professor, Experimental Hematology and Cancer Biology

Appointment

Adjunct Professor, Harvard Medical School

Email

david.williams@cchmc.org

Phone

513-636-0364

Bio

Dr. Williams’ major research interests are in the study of blood stem cell biology, blood formation, leukemia and the treatment of genetic blood diseases using gene therapy. He has published over 200 peer-reviewed manuscripts and textbook chapters. He was an investigator of the HHMI for 16 years. He has been continuously funded by the NIH since 1986. His laboratory co-discovered and cloned Interleukin-11 (Neumega™) and carried out developmental studies that assisted in obtaining FDA approval of this drug. His laboratory also described the use of fibronectin in retrovirus-mediated gene transfer, now nearly universally used (as Retronectin®) in clinical gene therapy trials and basic research utilizing gene transfer.

Dr. Williams basic research has focused on hematopoietic stem cell biology and specifically molecular and biochemical analysis of the interaction between hematopoietic stem cells and the bone marrow supporting environment. This work has recently focused on the roles of Rho GTPases in stem cell engraftment, retention and mobilization from the bone marrow. His laboratory has defined Rac GTPases (Rac1, Rac2 in particular) as key regulatory switches that control stem cell adhesion, migration and survival/proliferation in the adult bone marrow cavity and during fetal development in yolk sac, fetal liver and other embryonic sites. His laboratory has also recently demonstrated that Rac GTPases are essential signaling molecules in BCR-ABL oncogene transformation of hematopoietic stem cells. He and Dr. Yi Zheng (Cincinnati Children’s Hospital) have developed a new small molecule inhibitor of Rac GTPases that is being investigated as a molecular target in cancer therapies. His laboratory is investigating the role of several other Rho GTPases in normal and abnormal hematopoiesis.

Dr. Williams is currently the Chief, Division of Hematology/Oncology and Director of Clinical and Translational Research, Children’s Hospital Boston and the Leland Fikes Professor of Pediatrics, Harvard Medical School. Dr. Williams is also an adjunct faculty member at Cincinnati Children's.

Credentials

BA: Indiana State University, Terre Haute, IN, 1975
MD: Indiana University School of Medicine, Indianapolis, IN, 1979
Residency: Cincinnati Children's Hospital Medical Center, Cincinnati, OH, 1979-1982
Fellowships: Harvard Medical School, Boston, MA, 1982-1985; The Children's Hospital Boston, Boston, MA, 1982-1985

Research

Genetic manipulation of mammalian cells; hematopoietic stem cell biology

Research Grants and Contracts

Novel Molecular and Cellular Therapies in Fanconi Anemia, National Institutes of Health, Prinicpal Investigator, April 2006 - March 2010

Chemoresistance and Stem Cell Selection, National Institutes of Health, Principal Investigator, January 2006 - December 2010

Rac proteins in hematoppietic cell survival and function, National Institute of Diabetes and Digestive and Kidney Diseases, Prinical Investigator, Fecruary 2002 - June 2012

RhoH GTPase in Hematopoiesis and Cancer, National Institutes of Health, Prinicipal Investigator, May 2005 - April 2010

Cell Type and Stimulus-Specific Signaling Role of CDC42, National Institutes of Health, July 2006 - May 2011

Role of FA Protein Complexes in Hematopoiesis, National Institutes of Health, July 2005 - June 2010

Dbl-like regulators of small GTP-binding proteins, National Institute of General Medical Sciences, April 2005 - March 2010

University of Cincinnati Clinical Research Curriculum Award, National Institutes of Health, Co-Principal Investigator, December 2004 - November 2009

The Molecular Basis Society of Acute Myeloid Leukemia, Leukemia & Lymphoma, July 2005 - June 2010

Training Program in Cancer Therapeutics, National Cancer Institute, September 2006 - August 2011

Rac GTPases as Targets in Lymphomagenesis, National Institutes of Health, Februaru 2007 - January 2012

Inhibition of Rac GTPases in the Therapy of Chronic Myelogenous Leukemia, Department of Defense, April 2007 - March 2010

Rac1 and Rac2 Guanosine Triphosphotase in Erythroid Function and Differentiation, National Heart, Lung and Blood Institute, February 2008 - November 2012

Role of tumor necrosis factor in leukemogenesis, Leukemia & Lymphoma Society, July 2008 - June 2013

Regulation of Systemis RNA Interface in the Nematode C elegans, National Institutes of Health, January 2009 - December 2013

Pathophysiology of Human Blood Cells, National Heart, Lung and Blood Institute, Principal Investigator, July 1975 - June 2010

Publications, Most Recent

Akbar H, Cancelas JA, Chen L, Johnson JF, Williams DA and Zheng Y: “Genetic and pharmacological evidence that Rac1 GTPase is involved in regulation of platelet secretion and aggregation.”Journal of Thrombosis and Haemostasis 5 (8):1747-1755, 2007.

Ball CR, Pilz IH, Schmidt M, Fessler S, Williams DA, von Kalle C and Glimm H: “Stable differentiation and clonality of murine long-term hematopoiesis after extended reduced intensity selection for MGMT P140K transgene expression.” Blood 110 (6): 1779-1787. 2007.

Kelly P, Radtke S, von Kalle C, Balcik B, Bohn K, Mueller R, Schuesler T, Haren M, Reeves L, Cancelas J, Leemhuis T, Harris R, Auerbach A, Smith F, Davies S and Williams DA: “Stem Cell collection and genetic correction of Fanconi anemia hematopoietic stem cells.”Molecular Therapy 15 (1): 211-219, 2007.

Li J, Sejas DP, Zhang X, Qiu Y, Nattamai KJ, Rani R, Rathbun KR, Geiger H, Williams DA, Bagby GC and Pang Q: TNF-α induces leukemic clonal evolution ex vivo in Fanconi anemia group C stem cells, Journal of Clinical Investigation, 117(11): 3283–3295, 2007.

Mehta PA, Ileri T, Harris RE, Williams DA, Mo J, Smolarek T, Auerbach AD, Kelly P and Davies SM: “Chemotherapy for myeloid malignancy in children with Fanconi Anemia.Pediatric Blood and Cancer 48 (7): 668-672, 2007.

Sejas DP, Rani R, Qiu Y, Zhang X, Fagerlie SR, Nakano H, Williams DA and Pang Q: "Inflammatory reactive oxygen species-mediated hematopoietic suppression in Fanccdeficient mice.” Journal of Immunology 178(8):5277-87, 2007.

Sherafat-Kazemzadeh R, Mehta SN, Care MM, Williams DA and Rose SR: “Small Pituitary size in children with Fanconi Anemia.” Pediatric Blood and Cancer 49 (2): 166-170, 2007.

Thomas EK, Harris CE, Druker BJ, Zheng Y, Cancelas JA and Williams DA: “Rac guanosine triphosphatases represent integrating molecular therapeutic targets for BCR/ABL-induced myeloproliferative disease.” Cancer Cell, 12: 467-478, 2007.

Will E, Bailey J, Schuesler T, Balcik B, Burzynski B,Witte D, Layh-Schmitt G, von Kalle C, Baum C, Sorrentino BP, Wagner LM, Kelly P, Reeves L and Williams DA: “Importance of murine study design for testing toxicity of retroviral vectors in support of Phase I trials,”Molecular Therapy 15(4): 782-91, 2007.

Yang L, Wang L, Kalfa T, Cancelas JA, Mo J, Williams DA and Zheng Y: “Cdc42 critically regulates the balance between myelopoiesis and erythropoiesis,”Blood, 110(12): 3853-3861, 2007.

Zhang X, Sejas DP, Qiu Y, Williams DA and Pang Q: "Inflammatory reactive oxygen species promote and cooperate with Fanconi anemia mutation for hematopoietic senescence,” Journal of Cell Science 120 (9):1572-1583, 2007.

Chae H, Lee KE, Williams DA and Gu Y: Cross-talk between RhoH and Rac1 in regulation of actin cytoskeleton and chemotaxis of hematopoietic progenitor cells.Blood, 111(5): 2597-605, 2008.

Marsh RA, Lucky AW, Walsh TJ, Pachecco MC, Rinaldi MG, Mailler-Savage E, Puel A, Casanova JL, Blessing JJ, Fuluppi MD, Williams DA, Daines MO and Filipovich AD: Cutaneous Infection with Metarhizium Anisopliae in a Patient with Hypohidrotic Ectodermal Dysplasia and Immune Deficiency.The Pediatric Infectious Disease Journal, 27(3):283-284, 2008.

*Ghiaur G, Ferkowicz MJ, Milsom MD, Bailey J, Cancelas JA, Yoder MC and Williams DA: "Rac1 is essential for intra-embryonic hematopoiesis and for the initial seeding of fetal liver with definitive hematopoietic progenitor cells.” Blood, 111( 7): 3313-3321, 2008. *Plenary Paper. Commentaries by Dr. Donald Metcalf and Dr. Margaret H. Baron

Muller L, Milsom MD and Williams DA: “Rapid lentiviral transduction preserves engraftment potential of Fanca-/- hematopoietic stem cells.” Molecular Therapy, 16(6):1154-60, 2008.

Wu X, Xiaolin T, Joeng KS, Hilton MJ, Williams DA and Long F: "Rac1 activation and subsequent of β-catenin phosphorylation controls nuclear localization of β-catenin during canonical signaling.” Cell, 133:340-353, 2008.

Yamada Y, Cancelas JA, Sanchez-Aguilera A, Brandt EB, McBride M, Al-Moamen NJH, Finkelman FD, Williams DA and Rothenberg ME: “FIP1L1/PDGFRα synergizes with SCF to induce systemic mastocytosis in chronic eosinophilic leukemia/hypereosinophilic syndrome mice.” Blood, 112(6) 2500-2507, 2008.

Thornhill SI, Schambach A, Howe SJ, Ulaganathan M, Sebire NJ, Will E, Williams DA, Kinnon C, Baum C and Thrasher AJ: "Self-inactivating gammaretroviral vectors for gene therapy of x-linked severe combined immunodeficiency.” Molecular Therapy, 16(3): 590-598, 2008

Milsom MD, Jerabek-Willemsen M, Harris CE, Schambach A, Broun E, Bailey J, Jansen M, Schleimer D, Nattamai K, Wilhelm J, Watson A, Geiger H, Margison GP, Moritz T, Baum C, Thomale J and Williams DA: “Reciprocal relationship between very high O6- methylguanine-DNA methyltransferase P140K expression level and chemoprotection/selection of hematopoietic stem cells.” Cancer Research, 68(15) 6171- 6180, 2008.

Guo F, Cancelas JA, Hildeman D, Williams DA and Zheng Y: “Rac GTPase isoforms, Rac1 and Rac2, play a redundant and crucial role in T-cell development.” Blood, 112(5): 1767-1775, 2008.

Muller L, Schore E, Thomas E, Kim M, Cancelas J, Gu Y and Williams DA: “Rac Guanosine Triphosphates represent a potential target in AML.” Leukemia,(22) 1804- 1808, 2008.

Zhang X, Shang X, Wang L, Guo F, Murphy K, Kirby M, Kelly P, Reeves L, Williams DA, Zheng Y and Pang Q: “Defective homing is associated with altered CDC42 activity in cells from Fanconi anemia group A patients.” Blood, 112(5):1683-1686, 2008.

Hoskins E, Morris T, Higginbotham J, Spardy N, Cha E, Kelly P, Williams DA, Wikenheiser-Brokamp K, Duensing S, Wells S: “Fanconi anemia deficiency stimulates HPV-associated hyperplastic growth in organotypic epithelial raft culture.” Oncogene, 28, 674-685, 2009.

Milsom M, Schiedlmeier B, Bailey J, MiOk K, Li D, Jansen M, Ali A, Kirby M, Baum C, Fairbairn L and Williams DA: “Ectopic HOXB4 overcomes the inhibitory effect of tumor necrosis factor- α on Fanconi anemia hematopoietic stem and progenitor cells” , Blood [epublished ahead of print:blood-2008-09-180224v1], 2009.

Jones EE, Habash KB, Wise-Draper TM, Cash TA, Jansen M, Kelly P, Williams DA, Knudsen ES and Wells SI: “Coordinate regulation of Fanconi Anemia gene expression occurs through the Rb/E2F pathway.” Cancer Research, In Press, 2008

Gan B, Shain E, Jiang S, Sanchez-Aguilera A, Scott K, Chin L, Williams DA, Kwaitkowski D, DePinho D: “mTORC1 dependent and independent regulation of stem cell renewal, differentiation and mobilization.” PNAS, In Press, 2008.

Mueller L, Daley G, Williams DA: “Upping the ante: Recent Advances in direct reprogramming.”Molecular Therapy, In Press, 2009.

Cancelas JA, Williams DA: “Rho GTPases in hematopoietic stem cell function.” Current Opinion in Hematology, In Press, 2009.

Velu C, Porteous S, Bourdeau T, Xu H, Kazanjian A, Baktula AM, Jansen M, Williams DA, Gilks CB, Colmenares C and Grimes HL: “Epigenetic Control of Hematopoietic Stem Cell Fitness and Myelopoiesis.” Submitted, 2008.

Pannucci N, Sahay S, Thomas E, Chen R, Megjugorac N, Adams H, Rodriguez P, Fitpatrick E, Lagunoff D, Williams DA and Whitehead I: “Loss of binding to the xeroderma pigmentosum group B protein impairs p210 BCR/ABL leukemogenic activity in a murine model for chronic myelogenous leukemia.” Submitted, 2008.

Kalfa T, Pushkaran S, Cancelas J, Johnson J, Daria D, Geiger H, Williams DA and Zheng Y: “Rac GTPases Regulate Early Erythropoietic Expansion and Erythroblast Enucleation.” Submitted, 2008.

Chae D, Siefring J, Hildeman D, Gu Yand Williams DA: “RhoH regulates subcellular localization of ZAP-70 and Lck in T cell receptor signaling.” Submitted, 2008.

Ali A, Kirby M, Jansen M, Lach F, Schulte J, Singh T, Batish S, Auerbach A, Williams, DA and Meetei, R: “An unusual presentation of FANCL: Making the case for complementation analysis in all Fanconi anemia patients.” Submitted, 2008.

Smith F, Jansen M, Williams DA: “Modern Management of Fanconi Anemia.”, Submitted, 2008.

Milsom MD, Lee AW, Zheng Y, Williams DA, Cancelas JA: Fanca-/-Hematopoietic stem cells demonstrate a mobilization defect which can be overcome by administrator of the Rac inhibitor NSC23766., Submitted, 2008.

Rattman I, Sanchez-Aguilera A, Drew DZ, Muller L, Summey V, Lucas DM, Byrd JC, Croce CM, Gu Y, Cancelas JA, Johnston P, Moritz T, Williams DA: “Involvement of RhoH GTPase in the Development of B-Cell Chronic Lymphocytic Leukemia.”, Submitted, 2009

Presentations, Most Recent

Invited Speaker, Trends in Technology Series, “Stem Cell Research,” Cincinnati Alliance for Healthcare Marketing, Cincinnati, OH, January, 2007.

Invited Speaker and Participant, NIDDK Workshop, Inherited Bone Marrow Failure Syndromes: “Laboratory Diagnoses of Fanconi Anemia,” Washington, DC, January, 2007.

Invited Speaker, Neurodegeneration with Brain, Iron, Accumulation Disorders Association Fourth International Family Conference: “Stem Cells? Will They Play A Healing Role?,” Cincinnati, OH, May 2007.

Invited Speaker, Red Cell Gordon Conference: “Rho GTPases in Red Cell Development and Membrane Integrity,” Aussois, France, May, 2007.

Invited Speaker, Cell Biology Seminar Series: “Rho GTPases: Integrating Key Signals Critical for Lymphohematopoiesis",Memorial Sloan Kettering, New York, NY, June 2007.

Invited Speaker, Germany Society of Gene Therapy: “Long live(d) Rac and Rho.” Hamburg, Germany, July 2007.

Invited Speaker, Germany Society of Gene Therapy: “Live or let die: the case for chemoprotection and stem cell selection using gene transfer in malignant and genetic diseases,” Hamburg, Germany, July 2007.

Invited Speaker-Chalk Talk for Students, German Society of Gene Therapy: “Rho GTPases in Stem Cell Localization and Retention,” Heidelberg, Germany, July 2007.

Invited Speaker, Molecular Characteristics of Normal and Leukemia Stem Cells: “Targeting Rac GTPase in leukemia stem cells,” European Society of Hematology, Foz do Iguazu, Brazil, July 2007.

Invited Speaker, ATP Tennis Tournament, “New Approaches to Leukemia Treatment Targeting Leukemia Stem Cells, Leukemia Lymphoma Society”, Cincinnati, OH, July 2007.

Invited Speaker, Camp Sunshine FA Family Meeting, “Clinical Trials in FA,” Fanconi Anemia Research Fund, Casco, ME, August, 2007.

Invited Speaker, “Rho GTPases in Normal and Abnormal Blood Development,” Dartmouth Medical School, Hannover, New Hampshire, September, 2007.

Invited Speaker, IV Conference on Stem Cell Gene Therapy, “Too little, too late: the initial experience in FA Gene Therapy”, Thessaloniki, Greece, September, 2007.

Invited Speaker, School of Nursing and Health Professions Continuing Education Series, “Stem Cell Research: Use of Stem Cells to Treat Human Diseases,” Northern Kentucky University, Highland Heights, KY, September, 2007.

Invited Speaker, Transatlantic Gene Therapy Retreat, “Too little, too late: the initial experience in FA Gene Therapy,” London, October, 2007.

Invited Speaker, Nineteenth Annual Fanconi Anemia Research Fund Scientific Symposium, “Genetic Correction of the Fanconi Anemia Stem Cell Defect by Ectopic Delivery of HOXB4,” Chicago, IL, 2007.

Invited Participant and Panel Discussant, The 2nd Symposium of the Robert A. Good Immunology Society, Boston MA, November, 2007.

Invited Speaker and Participant, Stem Cell Day at Children’s Hospital hosted by the Harvard Stem Cell Institute, “GTPase regulation of stem cell migration during ontogeny”, Boston, MA, December, 2007.

Invited Speaker and Participant, NIDDK Hematology Research Program, Strategic Planning Workshop, January, 2008

Invited Speaker, Stem Cell Day at Children’s Hospital hosted by the Harvard Stem Cell Institute, “GTPase regulation of stem cell migration during ontogeny”, Boston, MA, December, 2007.

Invited Speaker, University of Louisville Stem Cell Institute Speaker Series, “Rho GTPases in normal and abnormal blood cell development”, Louisville, KY, January, 2008.

Invited Speaker , Distinguished Lecture Series at the Cancer Institute of New Jersey, “Rac and Rho H as novel molecular targets in chronic leukemias,” New Brunswick, NY, February, 2008.

Invited Speaker and Session Chair, American Society for Gene Therapy Annual Meeting, ”Hemopoietic: Hematopoietic Stem Cell Niche” Boston, MA, May, 2008.

Invited Speaker, 4th Stem Cell and Clonality & Genotoxicity Retreat, “Chemical and Insertional Genotoxicity – Fanconi Anemia and the Methylguanine DNA Methyl Transferase Trials”, Boston, MA, June 2008.

Invited Speaker and Session Chair, 9th FASEB Summer Research Conference on Regulation and Function of Small GTPases, “Rac and Rho H in normal and abnormal blood cell development and function,” Vermont Academy, Saxtons River, VT, July 2008.

Invited Speaker, Center for Genomic Regulation, “Rac and Rho: The Ying and Yang of Hematopoiesis,” Barcelona, Spain, July, 2008.

Invited Speaker and Session Organizer, 4th Annual Center for Human Cell Therapy- Genetic Modification Approaches for Cell Therapy Symposium, “Current Issues in Human Gene Therapy,”,Boston, MA, November 2008.

Invited Speaker, Innovations in Pediatric Medicine, “Molecular Characterization of Normal and Leukemic Stem Cells”,New York, NY, November 2008. Keynote Lecturer, European Society of Gene and Cell Therapy, “Hematopoietic stem cells: challenging targets for genetic modification,” Brugge, Belgium November, 2008.

Invited Speaker, Gene Therapy Tuesday Seminar Series,Baylor College of Medicine, "Fibronectin-facilitated gene transfer: translational studies leading to new understanding of hematopoietic stem cell engraftment, mobilization and leukemogenesis", Houston, TX, March 2009.

Invited Speaker, Sanquin Spring Seminars – Blood and Beyond, “Rho GTPases in the Regulation of Normal and Abnormal Hematopoietic Stem Cell Function”, Amsterdam, Netherlands, April 2009.

Abstracts

Lo A, Mitchell T, Abel M, Dooley J, Vliagoftis H, Williams DA, Rothenberg ME, Eitzen G, Zimmermann N, Lacy P: Eosinophil superoxide release and airway hyperresponsiveness are not dependent on Rac2 GTPase, International Eosinophil Society, 2007.

Balcik B, Leemhuis T, Reeves L, Murphy K, Wagner L, Williams DA: Clinical-scale transduction of a drug resistance gene into hematopoietic progenitor cells using a retroviral vector. American Society of Blood Banks Annual Meeting, 2007.

van der Loo JCM, Swaney WS, Nordling D, Baum C, Williams DA, Malik P, and Reeves L: Production of retroviral vectors by transfection in a closed system bioreactor. Presented at Viral Vectors and Vaccines WilBio Conferences, Austin, TX, 2007.

Spiteri E, Lach FP, Fang F, Sims AE, Huang TT, Hanenberg H, Schindler D, Williams DA, Auerbach AD: New FA Genes in IFAR patients from North America: 25 year update and genotype/phenotype correlations. Presented at Fanconi Anemia Research Fund Annual Meeting, Chicago IL, 2007.

Ali A, Burzynski BB, Kirby M, Grassman E, Lach FP, Auerbach A, Meetei RA, Cancelas JA, Williams DA, Jansen M: An usual presentation of FANCL: Making the case for complementation analysis in all FA patients. Presented at the Fanconi Anemia Research Fund Annual Meeting, Chicago, IL, 2007. *Best Translational Poster

Zhang X, Shang X, Wang L, Guo F, Kirby M, Murphy K, Reeves L, Williams DA, Zheng Y, Pang Q: Defective adhesion, migration and homing associated with altered Rho GTPase activity in cells from FA patients. Presented at the Fanconi Anemia Research Fund Annual Meeting, Chicago, IL, 2007. * Best Basic Science Poster

Muller L, Milsom M, Williams DA: A dual approach to enhance engraftment of Fanconi anemia HSC employing rapid transduction and in vivo selection. Presented at the Fanconi Anemia Research Fund Annual Meeting, Chicago, IL, 2007.

Rattman I, Williams DA, Moritz T: Transient in vivo enrichment of cytidine deaminase overexpressing hematopoiesis by extended low dose cytarabine treatment. Presented at the International Society of Experimental Hematology, Hamburg, Germany, 2007.

Schambach A, Galla M, Maetzig T, Zychlinski D, Modlich U, Milsom M, Grassman E, Mishra A, van der Loo H, Williams DA, Bailey S, Thrasher A, Baum C: Retroviral SIN vector platform: From basic biology to clinical application. Presented at the International Society of Experimental Hematology, Hamburg, Germany, 2007.

Muller L, Milsom M, Williams DA: A dual approach to enhance engraftment of Fanconi anemia HSC employing rapid transduction and in vivo selection. Presented at the International Society of Experimental Hematology, Hamburg, Germany, 2007.

Milsom M, Harris CE, Schambach A, Broun E, Bailey J, Baum C, Margison G, Rattman I, Jerabek-Willemsen M, Moritz T, Thomale J, Kim M, Grassman E, Williams DA: Very high levels of MGMT P140K mutant inhibit cellular proliferation and compromise engraftment in gene modified murine HSC. Presented at the International Society of Experimental Hematology, Hamburg, Germany, 2007.

Lo A, Mitchell T, Abel M, Dooley J, Vliagoftis H, Williams DA, Rothenberg ME, Eitzen G, Zimmerman N, Lacy P: The role of Rac2 in eosinophil superoxide release and allergic airway responses. Submitted to American Academy of Allergy, Asthma and Immunology, Philadelphia, PA, 2007

Rattmann I, Zhu D, Croce CM, Byrd JC, Gu Y, Moritz T, Williams DA: The Expression of the GTPase-Deficient, Hematopoietic-Specific RhoH GTPase Is Implicated in Development of Chronic Lymphocytic Leukemia (CLL). Presented at the American Society of Hematology Annual Meeting, Atlanta, GA, 2007

Xu H, Geiger H, Szczur K, Deira D, Zheng Y, Settleman J, Williams DA, Fillippi MD: Loss of the Rho GTPase Activating Protein p190-B Enhances Hematopoietic Stem Cell Engraftment Potential. Presented at the American Society of Hematology Annual Meeting, Atlanta, GA, 2007

Szczar K, Zheng Y, Filippi MD: Rho GTPase Cdc42 Maintains Neutrophil Polarity during Directed Migration by Regulating CD11b Integrin Signals. Presented at the American Society of Hematology Annual Meeting, Atlanta, GA, 2007

Muller, LUW, Milsom M, Harris CE, Bailey J, Williams DA: The Sooner the Better: Rapid Transduction Enhances the Engraftment/Selection of Gene Corrected Fanconi Anemia HSC. Presented at the American Society of Hematology Annual Meeting, Atlanta, GA, 2007

Milsom MD, Harris CE, Schambach A, Broun E, Jerabek-Willemsen M, Baum C, Moritz T, Margison G, Thomale J, Williams DA: Very High Expression of MGMT(P140K) Paradoxically Impairs Proliferation, In Vivo Selection and Protection of Transduced Hematopoietic Stem Cells. Presented at the American Society of Hematology Annual Meeting, Atlanta, GA, 2007

Thomas EK, Cancelas JA, Chae H, Cox AD, Keller PJ, Perrotti D, Neviani P, Druker BJ, Zheng Y, Harris, CE, Williams DA: Rac GTPases Are Potential Therapeutic Targets in p210-BCR-ABL-Induced Myeloproliferative Disease (MPD). Presented at the American Society of Hematology Annual Meeting, Atlanta, GA, 2007

Kalfa TA, Pushkaran S, Cancelas JA, Johnson JF, Daria D, Geiger H, Williams DA, Zheng Y: Rac GTPases Regulate Erythropoiesis Both in the Early Steps of Differentiation and in Enucleation. Presented at the American Society of Hematology Annual Meeting, Atlanta, GA, 2007

Li L, Murali B, Worsham DN, Dunn SK, Cancelas JA: Dissection of the Role of Connexin-43 in Hematopoietic Progenitors and Stromal Cells Unveils Distinct Hematopoietic Intrinsic and Extrinsic Regulatory Functions. Presented at the American Society of Hematology Annual Meeting, Atlanta, GA, 2007

Sanchez-Aguilera A, Cancelas JA, Williams DA: RhoH-Deficient Mice Show Altered B Cell Populations In Vivo. Presented at the American Society of Hematology Annual Meeting, Atlanta, GA, 2007

Chae HD, Siefring JE, Hildeman DA, Gu Y, Williams DA: RhoH Functions as an Adaptor Molecule for ZAP-70 in TCR Signaling Pathway. Presented at the American Society of Hematology Annual Meeting, Atlanta, GA, 2007

Milsom MD, Schiedlmeier B, Bailey J, Schambach A, Baum C, Kirby M, Ali A, Meetei R, Grassman E and Williams DA: Too little, too late: the initial experience in FA Gene Therapy. Presented at the IV Conference on Stem Cell Gene Therapy, Thessaloniki, Greece, September, 2007.

Yamada Y, Cancelas JA, Brandt EB, Sanchez-Aguilera A, McBride M, Al-Moamen NJH, Finkelman F, Williams DA, Rothenburg ME: FIP1L1/PDGFRa Synergizes with SCF/c- Kit Signaling To Induce Systemic Mastocytosis in a Chronic Eosinophilic Leukemia Murine Model. Presented at the American Society of Hematology Annual Meeting,
Atlanta, GA, 2007

Yamada Y, Cancelas J, Sanchez-Aguilera A, McBride M, Williams DA, Rothenberg, M: FIP1L1/PDGFRA (F/P) Fusion Gene Induces Eosinophil Development, Activation, and Survival Synergistically with SCF and IL-5. Presented at the American Academy of Allergy, Asthma, and Immunology Annual Meeting, Philadelphia, PA, 2008.

van der Loo JC, Swaney WP, Zeigler TE, Terwilliger AR, Nordling DL, Schambach A, Baum C, Williams DA, Malik P, Reeves L: “Production of High Titer GMP-Grade Retroviral Vectors by Transfection in a Closed System Bioreactor.” Presented at the American Society for Gene Therapy Annual Meeting, Boston, MA 2008. Molecular Therapy 16(1), S165, 2008.

Grassman E, Kaiser E, Balcik B, Hall D, Witte D, Thornhill SI, Schambach A, Thrasher AJ, Baum C: “Validation of Murine Safety Study Design in Support of Phase I Gene Transfer Trials”. Presented at the American Society for Gene Therapy Annual Meeting, Boston, MA 2008. Molecular Therapy 16(1): S234, 2008.

Terwilliger AR, Grassman E, Balcik B, Kaiser EM, Hall D, Schambach A, Williams DA, Reeves L, Malik P. “Transduction of Human CD34+ Hematopoietic Progenitor Cells Using Pulmozyme® Treated Retroviral Vector.” Presented at the American Society for Gene Therapy Annual Meeting, Boston, MA 2008. Molecular Therapy 16(1): S236,
2008.

Thornhill SI, Grassman E, Ulaganathan M, Waddington SN, Van Der Loo H, Howe SJ, Schambach A, Kinnon C, Baum C, Williams DA, Thrasher A: “Self-Inactivating Retroviral Vectors for Gene Therapy of SCID-X1” Presented at the American Society for Gene Therapy Annual Meeting, Boston, MA 2008. Molecular Therapy 16(1): S84, 2008.

Metha PA, Williams DA, Pang Q, Harris R, Mueller R, Smith FO, Davies SM: “Etanercept (Enbrel) in Children with Fanconi Anemia and Early Bone Marrow Failure; a Pilot Study” To be Presented at Fanconi Anemia Research Fund Scientific Symposium, Eugene, OR 2008.

Xu H, Geiger H, Szczur K, Deira D, Zheng Y, Settleman J, Srour E, Williams DA, Filippi, MD: “Absence of the Rho GTPase Activating Protein p190-B Enhances Long Term Hematopoietic Stem Cell Engraftment.” Presented at the American Society of Hematology 50th Annual Meeting, San Francisco, CA 2008.

Pannucci N, Sahay S, Thomas E, Chen R, Megjugorac N, Adams H, Rodriguez P, Fitzpatrick E, Williams DA, Whitehead I: “Xeroderma Pigmentosum Group B Binding Contributes to p210 BCR/ABL Mediated Transformation in CML.” Presented at the American Society of Hematology 50th Annual Meeting, San Francisco, CA 2008.

Zhang X, Shang X, Guo F, Murphy K, Kirby M, Kelly P, Reeves L, Smith F, Williams DA, Zheng Y, Pang Q: “A Cell-Autonomous Defect of Bone Marrow Hematopoietic Stem/Progenitor Cells and Impaired Stromal Function in FA-a Patients.” Presented at the American Society of Hematology 50th Annual Meeting, San Francisco, CA 2008.

Velu C, Berk M, Xu H, Bourdeau T, Kazanjian A, Williams DA, Colmenares C, Grimes L: “The Ski oncoprotein regulates Hematopoietic Stem Cell Fitness and Functions as a Corepressor for Gfil1.” Presented at the American Society of Hematology 50th Annual Meeting, San Francisco, CA 2008.

Milsom M, Schiedlmeier B, Bailey J, Ali A, Kim M, Baum C, Jansen M, Williams DA: “Ectopic HOXB4 Overcomes the Inhibitory Effect of TNFα on Hematopoietic Stem and Progenitor Cells in a Murine Fanconi Anemia Model.” Presented at the American Society of Hematology 50th Annual Meeting, San Francisco, CA 2008.

Wei J, Wunderlich M, Harris C, Mizukawa B, Zheng Y, Williams DA, Mulloy J: “Rac GTPases Are Required for MLL-AF9-Induced Mixed Lineage Leukemia.” Presented at the American Society of Hematology 50th Annual Meeting, San Francisco, CA 2008.

van der Loo J, Swaney W, Nordling D, Schambach A, Baum C, Williams DA, Reeves L, Malik P: “Production of High Titer cGMP-Grade SIN Gamma-Retroviral Vectors by Transfection in a Closed System Bioreactor.” Presented at the American Society of Hematology 50th Annual Meeting, San Francisco, CA 2008.

Sanchez-Aguilera A, Sengupta A, Mastin J, Chang K, Williams DA, Cancelas J: “Rac2 GTPase Activation Is Necessary for Development of p190-BCR-ABL-Induced B-Cell Acute Lymphoblastic Leukemia.” Presented at the American Society of Hematology 50th Annual Meeting, San Francisco, CA 2008.

Mizukawa B, Wei J, Wunderlich M, Chou F, Harris C, Zheng Y, Williams DA, Mulloy J: “Rac GTPases and Bcl-2 Survival Signals are required for MLL-AF9-Induced Mixed Lineage Leukemia.” Presented at 7th Midwest Blood Club Meeting, Cincinnati, OH, 2009.