EDUCATION

Medical School

  • Indiana University School of Medicine , 1979 , Indianapolis , IN

Internship

  • Cincinnati's Children's Hospital Medical Center , 1980 , Cincinnati , OH

Residency

  • Cincinnati's Children's Hospital Medical Center , 1982 , Cincinnati , OH

Fellowship

Hematology/Oncology
  • Boston Children's Hospital/Dana-Farber Cancer Institute , 1985 , Boston , MA

PROFESSIONAL HISTORY

Dr. David A. Williams is a graduate of Indiana State University and Indiana University School of Medicine. After training in Pediatrics at Cincinnati Children's Hospital Medical Center, Dr. Williams completed fellowship training in Pediatric Hematology/Oncology at Dana-Farber/Boston Children's. He completed his research training here and at the Whitehead Institute at the Massachusetts Institute of Technology. He then became a faculty member at Harvard Medical School and of the Howard Hughes Medical Institute (HHMI).


* Dr. Willliams is on leave from his role as Chief, Hematology/Oncology, from September 19, 2019 - August 20, 2020

CERTIFICATIONS

  • American Board of Pediatrics, General Pediatrics

PUBLICATIONS

Publications powered by Harvard Catalyst Profiles

  1. Mechanisms underlying genetic susceptibility to multisystem inflammatory syndrome in children (MIS-C). J Allergy Clin Immunol. 2021 09; 148(3):732-738.e1. View abstract
  2. Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease. N Engl J Med. 2021 01 21; 384(3):205-215. View abstract
  3. A study assessing the feasibility of randomization of pediatric and young adult patients between matched unrelated donor bone marrow transplantation and immune-suppressive therapy for newly diagnosed severe aplastic anemia: A joint pilot trial of the North American Pediatric Aplastic Anemia Consortium and the Pediatric Transplantation and Cellular Therapy Consortium. Pediatr Blood Cancer. 2020 10; 67(10):e28444. View abstract
  4. The Changing Face of Adrenoleukodystrophy. Endocr Rev. 2020 08 01; 41(4). View abstract
  5. Peripheral blood smears of children with multisystem inflammatory syndrome demonstrate prominence of early myeloid forms with morphologic evidence of toxic change. Pediatr Blood Cancer. 2021 01; 68(1):e28551. View abstract
  6. Children's rare disease cohorts: an integrative research and clinical genomics initiative. NPJ Genom Med. 2020; 5:29. View abstract
  7. Children's rare disease cohorts: an integrative research and clinical genomics initiative. NPJ Genom Med. 2020 Jul 06; 5(1):29. View abstract
  8. Current and future gene therapies for hemoglobinopathies. Curr Opin Hematol. 2020 05; 27(3):149-154. View abstract
  9. Infantile Myelofibrosis and Myeloproliferation with CDC42 Dysfunction. J Clin Immunol. 2020 05; 40(4):554-566. View abstract
  10. Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy. Mol Ther Methods Clin Dev. 2020 Jun 12; 17:589-600. View abstract
  11. Highly efficient therapeutic gene editing of human hematopoietic stem cells. Nat Med. 2019 05; 25(5):776-783. View abstract
  12. Two Decades of Molecular Therapy and a 35-Year Personal View of Changes in Gene Therapy. Mol Ther. 2019 03 06; 27(3):479-480. View abstract
  13. Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients. Blood Adv. 2018 10 09; 2(19):2505-2512. View abstract
  14. The pseudokinase MLKL activates PAD4-dependent NET formation in necroptotic neutrophils. Sci Signal. 2018 09 04; 11(546). View abstract
  15. DROSHA Knockout Leads to Enhancement of Viral Titers for Vectors Encoding miRNA-Adapted shRNAs. Mol Ther Nucleic Acids. 2018 Sep 07; 12:591-599. View abstract
  16. Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2018 02 01; 378(5):490-491. View abstract
  17. The gp130 Cytokine Interleukin-11 Regulates Engraftment of Vav1-/- Hematopoietic Stem and Progenitor Cells in Lethally Irradiated Recipients. Stem Cells. 2018 03; 36(3):446-457. View abstract
  18. A key role for Rac and Pak signaling in neutrophil extracellular traps (NETs) formation defines a new potential therapeutic target. Am J Hematol. 2018 02; 93(2):269-276. View abstract
  19. Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2017 10 26; 377(17):1630-1638. View abstract
  20. The 2017 ASPHO distinguished career award goes to Holcombe E. Grier, MD. Pediatr Blood Cancer. 2017 06; 64 Suppl 1. View abstract
  21. Evolving Gene Therapy in Primary Immunodeficiency. Mol Ther. 2017 05 03; 25(5):1132-1141. View abstract
  22. Rapid Lentiviral Transduction Preserves the Engraftment Potential of Fanca-/- Hematopoietic Stem Cells. Mol Ther. 2008 Jun; 16(6):1154-1160. View abstract
  23. Of Mouse Models and Men. Mol Ther. 2007 Apr; 15(4):643. View abstract
  24. AAV and Insertional Mutagenesis. Mol Ther. 2007 Oct; 15(10):1737. View abstract
  25. Molecular Therapy Moves on to a New Editor-in-Chief. Mol Ther. 2009 Dec; 17(12):1989-1990. View abstract
  26. Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype. J Clin Invest. 2016 10 03; 126(10):3868-3878. View abstract
  27. Assessment of physical function and participation in chronic pain clinical trials: IMMPACT/OMERACT recommendations. Pain. 2016 Sep; 157(9):1836-1850. View abstract
  28. RhoH participates in a multi-protein complex with the zinc finger protein kaiso that regulates both cytoskeletal structures and chemokine-induced T cells. Small GTPases. 2018 05 04; 9(3):260-273. View abstract
  29. Two Phase 3 Trials of Adalimumab for Hidradenitis Suppurativa. N Engl J Med. 2016 Aug 04; 375(5):422-34. View abstract
  30. Mathematical modeling of erythrocyte chimerism informs genetic intervention strategies for sickle cell disease. Am J Hematol. 2016 09; 91(9):931-7. View abstract
  31. The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice. Cancer Cell. 2016 07 11; 30(1):183. View abstract
  32. Mechanism of Drug-Drug Interactions Between Warfarin and Statins. J Pharm Sci. 2016 06; 105(6):1976-1986. View abstract
  33. The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice. Cancer Cell. 2016 04 11; 29(4):574-586. View abstract
  34. Response to correspondence: Flow cytometric quantification of neutrophil extracellular traps: Limitations of the methodological approach by Ciepiela et al. Am J Hematol. 2016 Mar; 91(3):E10. View abstract
  35. p21-activated kinase 2 regulates HSPC cytoskeleton, migration, and homing via CDC42 activation and interaction with ß-Pix. Blood. 2016 Apr 21; 127(16):1967-75. View abstract
  36. Comparison of enzyme kinetics of warfarin analyzed by LC-MS/MS QTrap and differential mobility spectrometry. J Chromatogr B Analyt Technol Biomed Life Sci. 2016 Jan 01; 1008:164-173. View abstract
  37. Flow cytometric assay for direct quantification of neutrophil extracellular traps in blood samples. Am J Hematol. 2015 Dec; 90(12):1155-8. View abstract
  38. miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction. Mol Ther. 2015 Sep; 23(9):1465-74. View abstract
  39. Telomerase inhibition effectively targets mouse and human AML stem cells and delays relapse following chemotherapy. Cell Stem Cell. 2014 Dec 04; 15(6):775-90. View abstract
  40. A modified ?-retrovirus vector for X-linked severe combined immunodeficiency. N Engl J Med. 2014 Oct 09; 371(15):1407-17. View abstract
  41. Unexpected help: mTOR meets lentiviral vectors. Blood. 2014 Aug 07; 124(6):832-3. View abstract
  42. Genetic deletion of the GATA1-regulated protein a-synuclein reduces oxidative stress and nitric oxide synthase levels in mature erythrocytes. Am J Hematol. 2014 Oct; 89(10):974-7. View abstract
  43. Charting a clear path: the ASGCT Standardized Pathways Conference. Mol Ther. 2014 Jul; 22(7):1235-1238. View abstract
  44. Loss of function of TET2 cooperates with constitutively active KIT in murine and human models of mastocytosis. PLoS One. 2014; 9(5):e96209. View abstract
  45. Concise review: lessons learned from clinical trials of gene therapy in monogenic immunodeficiency diseases. Stem Cells Transl Med. 2014 May; 3(5):636-42. View abstract
  46. Retroviral transduction of murine and human hematopoietic progenitors and stem cells. Methods Mol Biol. 2014; 1185:287-309. View abstract
  47. Diagnosis and treatment of pediatric acquired aplastic anemia (AAA): an initial survey of the North American Pediatric Aplastic Anemia Consortium (NAPAAC). Pediatr Blood Cancer. 2014 May; 61(5):869-74. View abstract
  48. A unique carboxyl-terminal insert domain in the hematopoietic-specific, GTPase-deficient Rho GTPase RhoH regulates post-translational processing. J Biol Chem. 2013 Dec 20; 288(51):36451-62. View abstract
  49. Oxandrolone for the treatment of bone marrow failure in Fanconi anemia. Pediatr Blood Cancer. 2014 Jan; 61(1):11-9. View abstract
  50. Broadening the indications for hematopoietic stem cell genetic therapies. Cell Stem Cell. 2013 Sep 05; 13(3):263-4. View abstract
  51. Depletion of Jak2V617F myeloproliferative neoplasm-propagating stem cells by interferon-a in a murine model of polycythemia vera. Blood. 2013 May 02; 121(18):3692-702. View abstract
  52. The Rac GTPase effector p21-activated kinase is essential for hematopoietic stem/progenitor cell migration and engraftment. Blood. 2013 Mar 28; 121(13):2474-82. View abstract
  53. Enough is indeed enough: ACGME required changes in pediatric training. Pediatr Blood Cancer. 2012 Dec 15; 59(7):1158-9. View abstract
  54. Induced pluripotent stem cells as a tool for gaining new insights into Fanconi anemia. Cell Cycle. 2012 Aug 15; 11(16):2985-90. View abstract
  55. CTC1 Mutations in a patient with dyskeratosis congenita. Pediatr Blood Cancer. 2012 Aug; 59(2):311-4. View abstract
  56. RhoH is critical for cell-microenvironment interactions in chronic lymphocytic leukemia in mice and humans. Blood. 2012 May 17; 119(20):4708-18. View abstract
  57. Signaling and cytoskeletal requirements in erythroblast enucleation. Blood. 2012 Jun 21; 119(25):6118-27. View abstract
  58. Overcoming reprogramming resistance of Fanconi anemia cells. Blood. 2012 Jun 07; 119(23):5449-57. View abstract
  59. In vivo enrichment of cytidine deaminase gene-modified hematopoietic cells by prolonged cytosine-arabinoside application. Cytotherapy. 2012 Apr; 14(4):451-60. View abstract
  60. Rac signaling in osteoblastic cells is required for normal bone development but is dispensable for hematopoietic development. Blood. 2012 Jan 19; 119(3):736-44. View abstract
  61. PTEN negatively regulates engulfment of apoptotic cells by modulating activation of Rac GTPase. J Immunol. 2011 Dec 01; 187(11):5783-94. View abstract
  62. Inhibition of Rac GTPase signaling and downstream prosurvival Bcl-2 proteins as combination targeted therapy in MLL-AF9 leukemia. Blood. 2011 Nov 10; 118(19):5235-45. View abstract
  63. Differential niche and Wnt requirements during acute myeloid leukemia progression. Blood. 2011 Sep 08; 118(10):2849-56. View abstract
  64. Guanine nucleotide exchange factor Vav1 regulates perivascular homing and bone marrow retention of hematopoietic stem and progenitor cells. Proc Natl Acad Sci U S A. 2011 Jun 07; 108(23):9607-12. View abstract
  65. Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting. Mol Ther. 2011 Jul; 19(7):1193-8. View abstract
  66. Transatlantic consortium spotlights need for changes in gene therapy trials. Mol Ther. 2010 Nov; 18(11):1892. View abstract
  67. Training program in cancer and blood diseases: Pediatric Hematology/Oncology Fellowship Program, Children's Hospital Boston/Dana-Farber Cancer Institute. Am J Hematol. 2010 Oct; 85(10):793-4. View abstract
  68. Gaining the hard yard: pre-clinical evaluation of lentiviral-mediated gene therapy for the treatment of beta-thalassemia. EMBO Mol Med. 2010 Aug; 2(8):291-3. View abstract
  69. The American Society of Pediatric Hematology/Oncology (ASPHO) 2010 Distinguished Career Award Goes to Dr. Sam Lux. Pediatr Blood Cancer. 2010 Jun; 54(6):785-6. View abstract
  70. The Apc(min) mouse has altered hematopoietic stem cell function and provides a model for MPD/MDS. Blood. 2010 Apr 29; 115(17):3489-97. View abstract
  71. Rac1 and Rac2 GTPases are necessary for early erythropoietic expansion in the bone marrow but not in the spleen. Haematologica. 2010 Jan; 95(1):27-35. View abstract
  72. Rac GTPases in human diseases. Dis Markers. 2010; 29(3-4):177-87. View abstract
  73. Hematology grants workshop. Hematology Am Soc Hematol Educ Program. 2010; 2010:189-90. View abstract
  74. Correction and apology. Mol Ther. 2009 Oct; 17(10):1660. View abstract
  75. National Institutes of Health releases new guidelines on human stem cell research. Mol Ther. 2009 Sep; 17(9):1485-6. View abstract
  76. Gene therapy continues to mature and to face challenges. Mol Ther. 2009 Aug; 17(8):1305-6. View abstract
  77. Rapid development of pluripotent stem cells as a potential therapeutic modality. Mol Ther. 2009 Jun; 17(6):929-30. View abstract
  78. Recombinant DNA advisory committee updates recommendations on gene transfer for x-linked severe combined immunodeficiency. Mol Ther. 2009 May; 17(5):751-2. View abstract
  79. Finding the needle in the hay stack: hematopoietic stem cells in Fanconi anemia. Mutat Res. 2009 Jul 31; 668(1-2):141-9. View abstract
  80. Progress in genetic therapy for severe combined immunodeficiency associated with adenosine deaminase deficiency. Mol Ther. 2009 Apr; 17(4):577-8. View abstract
  81. Upping the ante: recent advances in direct reprogramming. Mol Ther. 2009 Jun; 17(6):947-53. View abstract
  82. Ectopic HOXB4 overcomes the inhibitory effect of tumor necrosis factor-{alpha} on Fanconi anemia hematopoietic stem and progenitor cells. Blood. 2009 May 21; 113(21):5111-20. View abstract
  83. New approaches in the potential treatment of HIV-acquired immunodeficiency disease. Mol Ther. 2009 Feb; 17(2):209-10. View abstract
  84. ESCGT 2008: progress in clinical gene therapy. Mol Ther. 2009 Jan; 17(1):1-2. View abstract
  85. mTORC1-dependent and -independent regulation of stem cell renewal, differentiation, and mobilization. Proc Natl Acad Sci U S A. 2008 Dec 09; 105(49):19384-9. View abstract
  86. Sleeping beauty vector system moves toward human trials in the United States. Mol Ther. 2008 Sep; 16(9):1515-6. View abstract
  87. Progress reported in two studies of clinical gene transfer into the retina. Mol Ther. 2008 Jul; 16(7):1181. View abstract
  88. Rac GTPase isoforms Rac1 and Rac2 play a redundant and crucial role in T-cell development. Blood. 2008 Sep 01; 112(5):1767-75. View abstract
  89. A "vector drain" in US gene therapy development? Mol Ther. 2008 May; 16(5):801-2. View abstract
  90. Rapid lentiviral transduction preserves the engraftment potential of Fanca(-/-) hematopoietic stem cells. Mol Ther. 2008 Jun; 16(6):1154-60. View abstract
  91. Foamy virus vectors come of age. Mol Ther. 2008 Apr; 16(4):635-6. View abstract
  92. NIH recombinant DNA Advisory Committee continues to ponder adverse event associated with AAV gene therapy trial. Mol Ther. 2008 Mar; 16(3):427-8. View abstract
  93. Rho GTPases and regulation of hematopoietic stem cell localization. Methods Enzymol. 2008; 439:365-93. View abstract
  94. Rac1 is essential for intraembryonic hematopoiesis and for the initial seeding of fetal liver with definitive hematopoietic progenitor cells. Blood. 2008 Apr 01; 111(7):3313-21. View abstract
  95. An international conversation on Stem Cell Gene Therapy. 4th Stem Cell Conference on Stem Cell Gene Therapy, Thessaloniki, Greece, 13-17 September 2007. Mol Ther. 2007 Dec; 15(12):2058-9. View abstract
  96. RAC reviews serious adverse event associated with AAV therapy trial. Mol Ther. 2007 Dec; 15(12):2053-4. View abstract
  97. Rac guanosine triphosphatases represent integrating molecular therapeutic targets for BCR-ABL-induced myeloproliferative disease. Cancer Cell. 2007 Nov; 12(5):467-78. View abstract
  98. Small pituitary size in children with Fanconi anemia. Pediatr Blood Cancer. 2007 Aug; 49(2):166-70. View abstract
  99. NIH decides against continuing NGVLs in their current form. Mol Ther. 2007 Jul; 15(7):1223. View abstract
  100. Chemotherapy for myeloid malignancy in children with Fanconi anemia. Pediatr Blood Cancer. 2007 Jun 15; 48(7):668-72. View abstract
  101. Live and let die: in vivo selection of gene-modified hematopoietic stem cells via MGMT-mediated chemoprotection. DNA Repair (Amst). 2007 Aug 01; 6(8):1210-21. View abstract
  102. Are lentivirus vectors safer? Mol Ther. 2007 Mar; 15(3):439. View abstract
  103. Importance of murine study design for testing toxicity of retroviral vectors in support of phase I trials. Mol Ther. 2007 Apr; 15(4):782-91. View abstract
  104. Stem cell collection and gene transfer in Fanconi anemia. Mol Ther. 2007 Jan; 15(1):211-9. View abstract
  105. ASGT advises NIH on funding of gene therapy trials. Mol Ther. 2007 Jan; 15(1):1-2. View abstract
  106. NIH funding of gene therapy trials. Mol Ther. 2006 Nov; 14(5):607. View abstract
  107. Vector insertion, mutagenesis and transgene toxicity. Mol Ther. 2006 Oct; 14(4):457. View abstract
  108. Rac GTPases regulate the morphology and deformability of the erythrocyte cytoskeleton. Blood. 2006 Dec 01; 108(12):3637-45. View abstract
  109. A pox on your tumor. Mol Ther. 2006 Sep; 14(3):313. View abstract
  110. Inhibition of RhoA GTPase activity enhances hematopoietic stem and progenitor cell proliferation and engraftment. Blood. 2006 Sep 15; 108(6):2087-94. View abstract
  111. Gene therapy advances but struggles to interpret safety data in small animal models. Mol Ther. 2006 Jun; 13(6):1027-8. View abstract
  112. Adventitious mutations in clinical grade vectors: an issue to consider? Mol Ther. 2006 May; 13(5):831-2. View abstract
  113. An emerging consensus on recommendations to facilitate clinical gene transfer. Mol Ther. 2006 Apr; 13(4):637-8. View abstract
  114. Determination of methenamine, methenamine mandelate and methenamine hippurate in pharmaceutical preparations using ion-exchange HPLC. J Pharm Biomed Anal. 2006 Mar 18; 40(5):1243-8. View abstract
  115. Simultaneous determination of triamcinolone acetonide and oxymetazoline hydrochloride in nasal spray formulations by HPLC. J Pharm Biomed Anal. 2006 Mar 18; 40(5):1273-80. View abstract
  116. New AAV serotypes may broaden the therapeutic pipeline to human gene therapy. Mol Ther. 2006 Jan; 13(1):1-2. View abstract
  117. FDA guidance document on monitoring delayed adverse events a good first start. Mol Ther. 2005 Dec; 12(6):991-2. View abstract
  118. Promoting translational research in academic health centers: navigating the "roadmap". Acad Med. 2005 Nov; 80(11):1012-8. View abstract
  119. Equal potency of gammaretroviral and lentiviral SIN vectors for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells. Mol Ther. 2006 Feb; 13(2):391-400. View abstract
  120. Gene therapy in the treatment of Fanconi anemia, a progressive bone marrow failure syndrome. Curr Opin Mol Ther. 2005 Oct; 7(5):461-6. View abstract
  121. A rapid method for retrovirus-mediated identification of complementation groups in Fanconi anemia patients. Mol Ther. 2005 Nov; 12(5):976-84. View abstract
  122. A balanced decision? Regulatory reaction to the "third case". Mol Ther. 2005 Jun; 11(6):819-20. View abstract
  123. The NIH roadmap: timing is everything. Mol Ther. 2005 Feb; 11(2):173. View abstract
  124. Real-Time PCR: an Effective Tool for Measuring Transduction Efficiency in Human Hematopoietic Progenitor Cells. Mol Ther. 2005 Mar; 11(3):483-491. View abstract
  125. Chance or necessity? Insertional mutagenesis in gene therapy and its consequences. Mol Ther. 2004 Jan; 9(1):5-13. View abstract
  126. Methylguanine methyltransferase-mediated in vivo selection and chemoprotection of allogeneic stem cells in a large-animal model. J Clin Invest. 2003 Nov; 112(10):1581-8. View abstract
  127. Medicine. Gene therapy--new challenges ahead. Science. 2003 Oct 17; 302(5644):400-1. View abstract
  128. A rare complex DNA rearrangement in the murine Steel gene results in exon duplication and a lethal phenotype. Blood. 2003 Nov 15; 102(10):3548-55. View abstract
  129. Side effects of retroviral gene transfer into hematopoietic stem cells. Blood. 2003 Mar 15; 101(6):2099-114. View abstract
  130. Gene Therapy 2000. Hematology Am Soc Hematol Educ Program. 2000; 376-393. View abstract
  131. Mouse model of X-linked chronic granulomatous disease, an inherited defect in phagocyte superoxide production. Nat Genet. 1995 Feb; 9(2):202-9. View abstract
  132. Novel human oncogene lbc detected by transfection with distinct homology regions to signal transduction products. Oncogene. 1994 Feb; 9(2):621-8. View abstract
  133. Long-term in vivo expression of a murine adenosine deaminase gene in rhesus monkey hematopoietic cells of multiple lineages after retroviral mediated gene transfer into CD34+ bone marrow cells. Blood. 1993 Oct 01; 82(7):1975-80. View abstract
  134. Restriction of neuroblastoma to the prostate gland in transgenic mice. Mol Cell Biol. 1991 Sep; 11(9):4518-27. View abstract
  135. Retroviral gene transfer of human adenosine deaminase in murine hematopoietic cells: effect of selectable marker sequences on long-term expression. Blood. 1991 Jul 15; 78(2):310-7. View abstract
  136. Gene transfer into murine hematopoietic stem cells and bone marrow stromal cells. Ann N Y Acad Sci. 1990; 612:398-406. View abstract
  137. Long-term expression of human adenosine deaminase in mice transplanted with retrovirus-infected hematopoietic stem cells. Proc Natl Acad Sci U S A. 1989 Nov; 86(22):8892-6. View abstract
  138. Gene therapy of somatic cells: status and prospects. Prog Med Genet. 1988; 7:130-42. View abstract
  139. Retrovirus-mediated gene transfer of human adenosine deaminase: expression of functional enzyme in murine hematopoietic stem cells in vivo. Mol Cell Biol. 1987 Oct; 7(10):3459-65. View abstract
  140. Transfer and expression of human ADA in murine hematopoietic stem cells. Prog Clin Biol Res. 1987; 251:567-80. View abstract
  141. Retrovirus-mediated transfer of human adenosine deaminase gene sequences into cells in culture and into murine hematopoietic cells in vivo. Proc Natl Acad Sci U S A. 1986 Apr; 83(8):2566-70. View abstract
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