Current Environment: Production

David A. Williams | 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

David A. Williams | Certifications

  • American Board of Pediatrics (General)

David A. Williams | 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).

David A. Williams | Media

Research

Gene therapy for sickle cell disease: The journey to a new treatment

Award

Dr. David Williams is the recipient of the 2023 ASGCT Founder’s Award

David A. Williams | Publications

  1. UM171 enhances fitness and engraftment of gene-modified hematopoietic stem cells from patients with sickle cell disease. Blood Adv. 2024 Nov 26; 8(22):5885-5895. View UM171 enhances fitness and engraftment of gene-modified hematopoietic stem cells from patients with sickle cell disease. Abstract

  2. Regulated GATA1 expression as a universal gene therapy for Diamond-Blackfan anemia. Cell Stem Cell. 2024 Nov 08. View Regulated GATA1 expression as a universal gene therapy for Diamond-Blackfan anemia. Abstract

  3. Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2024 Oct 10; 391(14):1287-1301. View Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy. Abstract

  4. Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2024 Oct 10; 391(14):1302-1312. View Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy. Abstract

  5. A novel high-titer, bifunctional lentiviral vector for autologous hematopoietic stem cell gene therapy of sickle cell disease. Mol Ther Methods Clin Dev. 2024 Jun 13; 32(2):101254. View A novel high-titer, bifunctional lentiviral vector for autologous hematopoietic stem cell gene therapy of sickle cell disease. Abstract

  6. Clonal selection of hematopoietic stem cells after gene therapy for sickle cell disease. Nat Med. 2023 Dec; 29(12):3175-3183. View Clonal selection of hematopoietic stem cells after gene therapy for sickle cell disease. Abstract

  7. Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome. Blood. 2023 10 12; 142(15):1281-1296. View Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome. Abstract

  8. Hematopoietic stem-cell gene therapy is associated with restored white matter microvascular function in cerebral adrenoleukodystrophy. Nat Commun. 2023 04 05; 14(1):1900. View Hematopoietic stem-cell gene therapy is associated with restored white matter microvascular function in cerebral adrenoleukodystrophy. Abstract

  9. Replication competent retrovirus testing (RCR) in the National Gene Vector Biorepository: No evidence of RCR in 1,595 post-treatment peripheral blood samples obtained from 60 clinical trials. Mol Ther. 2023 03 01; 31(3):801-809. View Replication competent retrovirus testing (RCR) in the National Gene Vector Biorepository: No evidence of RCR in 1,595 post-treatment peripheral blood samples obtained from 60 clinical trials. Abstract

  10. Transient neonatal hemolytic anemia due to the novel gamma globin gene mutation HBG2:C.290T>C, p.Leu97Pro (hemoglobin Wareham). Pediatr Blood Cancer. 2023 01; 70(1):e30067. View Transient neonatal hemolytic anemia due to the novel gamma globin gene mutation HBG2:C.290T>C, p.Leu97Pro (hemoglobin Wareham). Abstract

  11. The long road traveled in hematopoietic stem cell gene therapy. Mol Ther. 2022 10 05; 30(10):3097-3099. View The long road traveled in hematopoietic stem cell gene therapy. Abstract

  12. Development of a double shmiR lentivirus effectively targeting both BCL11A and ZNF410 for enhanced induction of fetal hemoglobin to treat ß-hemoglobinopathies. Mol Ther. 2022 08 03; 30(8):2693-2708. View Development of a double shmiR lentivirus effectively targeting both BCL11A and ZNF410 for enhanced induction of fetal hemoglobin to treat ß-hemoglobinopathies. Abstract

  13. Validation of a small molecule inhibitor of PDE6D-RAS interaction with favorable anti-leukemic effects. Blood Cancer J. 2022 04 14; 12(4):64. View Validation of a small molecule inhibitor of PDE6D-RAS interaction with favorable anti-leukemic effects. Abstract

  14. Clonal hematopoiesis in sickle cell disease. J Clin Invest. 2022 02 15; 132(4). View Clonal hematopoiesis in sickle cell disease. Abstract

  15. Deletion of murine Rhoh leads to de-repression of Bcl-6 via decreased KAISO levels and accelerates a malignancy phenotype in a murine model of lymphoma. Small GTPases. 2022 01; 13(1):267-281. View Deletion of murine Rhoh leads to de-repression of Bcl-6 via decreased KAISO levels and accelerates a malignancy phenotype in a murine model of lymphoma. Abstract

  16. Investigational curative gene therapy approaches to sickle cell disease. Blood Adv. 2021 12 14; 5(23):5452. View Investigational curative gene therapy approaches to sickle cell disease. Abstract

  17. Mechanisms underlying genetic susceptibility to multisystem inflammatory syndrome in children (MIS-C). J Allergy Clin Immunol. 2021 09; 148(3):732-738.e1. View Mechanisms underlying genetic susceptibility to multisystem inflammatory syndrome in children (MIS-C). Abstract

  18. Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease. N Engl J Med. 2021 01 21; 384(3):205-215. View Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease. Abstract

  19. 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 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. Abstract

  20. The Changing Face of Adrenoleukodystrophy. Endocr Rev. 2020 08 01; 41(4). View The Changing Face of Adrenoleukodystrophy. Abstract

  21. 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 Peripheral blood smears of children with multisystem inflammatory syndrome demonstrate prominence of early myeloid forms with morphologic evidence of toxic change. Abstract

  22. Children's rare disease cohorts: an integrative research and clinical genomics initiative. NPJ Genom Med. 2020; 5:29. View Children's rare disease cohorts: an integrative research and clinical genomics initiative. Abstract

  23. Children's rare disease cohorts: an integrative research and clinical genomics initiative. NPJ Genom Med. 2020 Jul 06; 5(1):29. View Children's rare disease cohorts: an integrative research and clinical genomics initiative. Abstract

  24. Current and future gene therapies for hemoglobinopathies. Curr Opin Hematol. 2020 05; 27(3):149-154. View Current and future gene therapies for hemoglobinopathies. Abstract

  25. Infantile Myelofibrosis and Myeloproliferation with CDC42 Dysfunction. J Clin Immunol. 2020 05; 40(4):554-566. View Infantile Myelofibrosis and Myeloproliferation with CDC42 Dysfunction. Abstract

  26. 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 Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy. Abstract

  27. Highly efficient therapeutic gene editing of human hematopoietic stem cells. Nat Med. 2019 May; 25(5):776-783. View Highly efficient therapeutic gene editing of human hematopoietic stem cells. Abstract

  28. 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 Two Decades of Molecular Therapy and a 35-Year Personal View of Changes in Gene Therapy. Abstract

  29. 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 Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients. Abstract

  30. The pseudokinase MLKL activates PAD4-dependent NET formation in necroptotic neutrophils. Sci Signal. 2018 09 04; 11(546). View The pseudokinase MLKL activates PAD4-dependent NET formation in necroptotic neutrophils. Abstract

  31. 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 DROSHA Knockout Leads to Enhancement of Viral Titers for Vectors Encoding miRNA-Adapted shRNAs. Abstract

  32. Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2018 02 01; 378(5):490-491. View Gene Therapy for Cerebral Adrenoleukodystrophy. Abstract

  33. 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 The gp130 Cytokine Interleukin-11 Regulates Engraftment of Vav1-/- Hematopoietic Stem and Progenitor Cells in Lethally Irradiated Recipients. Abstract

  34. 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 A key role for Rac and Pak signaling in neutrophil extracellular traps (NETs) formation defines a new potential therapeutic target. Abstract

  35. Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2017 10 26; 377(17):1630-1638. View Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy. Abstract

  36. The 2017 ASPHO distinguished career award goes to Holcombe E. Grier, MD. Pediatr Blood Cancer. 2017 06; 64 Suppl 1. View The 2017 ASPHO distinguished career award goes to Holcombe E. Grier, MD. Abstract

  37. Evolving Gene Therapy in Primary Immunodeficiency. Mol Ther. 2017 05 03; 25(5):1132-1141. View Evolving Gene Therapy in Primary Immunodeficiency. Abstract

  38. Rapid Lentiviral Transduction Preserves the Engraftment Potential of Fanca-/- Hematopoietic Stem Cells. Mol Ther. 2008 Jun; 16(6):1154-1160. View Rapid Lentiviral Transduction Preserves the Engraftment Potential of Fanca-/- Hematopoietic Stem Cells. Abstract

  39. AAV and Insertional Mutagenesis. Mol Ther. 2007 Oct; 15(10):1737. View AAV and Insertional Mutagenesis. Abstract

  40. Of Mouse Models and Men. Mol Ther. 2007 Apr; 15(4):643. View Of Mouse Models and Men. Abstract

  41. Molecular Therapy Moves on to a New Editor-in-Chief. Mol Ther. 2009 Dec; 17(12):1989-1990. View Molecular Therapy Moves on to a New Editor-in-Chief. Abstract

  42. Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype. J Clin Invest. 2016 10 03; 126(10):3868-3878. View Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype. Abstract

  43. Assessment of physical function and participation in chronic pain clinical trials: IMMPACT/OMERACT recommendations. Pain. 2016 Sep; 157(9):1836-1850. View Assessment of physical function and participation in chronic pain clinical trials: IMMPACT/OMERACT recommendations. Abstract

  44. 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 RhoH participates in a multi-protein complex with the zinc finger protein kaiso that regulates both cytoskeletal structures and chemokine-induced T cells. Abstract

  45. Two Phase 3 Trials of Adalimumab for Hidradenitis Suppurativa. N Engl J Med. 2016 Aug 04; 375(5):422-34. View Two Phase 3 Trials of Adalimumab for Hidradenitis Suppurativa. Abstract

  46. Mathematical modeling of erythrocyte chimerism informs genetic intervention strategies for sickle cell disease. Am J Hematol. 2016 09; 91(9):931-7. View Mathematical modeling of erythrocyte chimerism informs genetic intervention strategies for sickle cell disease. Abstract

  47. The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice. Cancer Cell. 2016 07 11; 30(1):183. View The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice. Abstract

  48. Mechanism of Drug-Drug Interactions Between Warfarin and Statins. J Pharm Sci. 2016 06; 105(6):1976-1986. View Mechanism of Drug-Drug Interactions Between Warfarin and Statins. Abstract

  49. 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 The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice. Abstract

  50. 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 p21-activated kinase 2 regulates HSPC cytoskeleton, migration, and homing via CDC42 activation and interaction with ß-Pix. Abstract

  51. 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 Response to correspondence: Flow cytometric quantification of neutrophil extracellular traps: Limitations of the methodological approach by Ciepiela et al. Abstract

  52. 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 Comparison of enzyme kinetics of warfarin analyzed by LC-MS/MS QTrap and differential mobility spectrometry. Abstract

  53. Flow cytometric assay for direct quantification of neutrophil extracellular traps in blood samples. Am J Hematol. 2015 Dec; 90(12):1155-8. View Flow cytometric assay for direct quantification of neutrophil extracellular traps in blood samples. Abstract

  54. miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction. Mol Ther. 2015 Sep; 23(9):1465-74. View miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction. Abstract

  55. 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 Telomerase inhibition effectively targets mouse and human AML stem cells and delays relapse following chemotherapy. Abstract

  56. A modified ?-retrovirus vector for X-linked severe combined immunodeficiency. N Engl J Med. 2014 Oct 09; 371(15):1407-17. View A modified ?-retrovirus vector for X-linked severe combined immunodeficiency. Abstract

  57. Unexpected help: mTOR meets lentiviral vectors. Blood. 2014 Aug 07; 124(6):832-3. View Unexpected help: mTOR meets lentiviral vectors. Abstract

  58. 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 Genetic deletion of the GATA1-regulated protein a-synuclein reduces oxidative stress and nitric oxide synthase levels in mature erythrocytes. Abstract

  59. Charting a clear path: the ASGCT Standardized Pathways Conference. Mol Ther. 2014 Jul; 22(7):1235-1238. View Charting a clear path: the ASGCT Standardized Pathways Conference. Abstract

  60. Loss of function of TET2 cooperates with constitutively active KIT in murine and human models of mastocytosis. PLoS One. 2014; 9(5):e96209. View Loss of function of TET2 cooperates with constitutively active KIT in murine and human models of mastocytosis. Abstract

  61. 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 Concise review: lessons learned from clinical trials of gene therapy in monogenic immunodeficiency diseases. Abstract

  62. Retroviral transduction of murine and human hematopoietic progenitors and stem cells. Methods Mol Biol. 2014; 1185:287-309. View Retroviral transduction of murine and human hematopoietic progenitors and stem cells. Abstract

  63. 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 Diagnosis and treatment of pediatric acquired aplastic anemia (AAA): an initial survey of the North American Pediatric Aplastic Anemia Consortium (NAPAAC). Abstract

  64. 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 A unique carboxyl-terminal insert domain in the hematopoietic-specific, GTPase-deficient Rho GTPase RhoH regulates post-translational processing. Abstract

  65. Oxandrolone for the treatment of bone marrow failure in Fanconi anemia. Pediatr Blood Cancer. 2014 Jan; 61(1):11-9. View Oxandrolone for the treatment of bone marrow failure in Fanconi anemia. Abstract

  66. Broadening the indications for hematopoietic stem cell genetic therapies. Cell Stem Cell. 2013 Sep 05; 13(3):263-4. View Broadening the indications for hematopoietic stem cell genetic therapies. Abstract

  67. 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 Depletion of Jak2V617F myeloproliferative neoplasm-propagating stem cells by interferon-a in a murine model of polycythemia vera. Abstract

  68. 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 The Rac GTPase effector p21-activated kinase is essential for hematopoietic stem/progenitor cell migration and engraftment. Abstract

  69. Enough is indeed enough: ACGME required changes in pediatric training. Pediatr Blood Cancer. 2012 Dec 15; 59(7):1158-9. View Enough is indeed enough: ACGME required changes in pediatric training. Abstract

  70. Induced pluripotent stem cells as a tool for gaining new insights into Fanconi anemia. Cell Cycle. 2012 Aug 15; 11(16):2985-90. View Induced pluripotent stem cells as a tool for gaining new insights into Fanconi anemia. Abstract

  71. CTC1 Mutations in a patient with dyskeratosis congenita. Pediatr Blood Cancer. 2012 Aug; 59(2):311-4. View CTC1 Mutations in a patient with dyskeratosis congenita. Abstract

  72. RhoH is critical for cell-microenvironment interactions in chronic lymphocytic leukemia in mice and humans. Blood. 2012 May 17; 119(20):4708-18. View RhoH is critical for cell-microenvironment interactions in chronic lymphocytic leukemia in mice and humans. Abstract

  73. Signaling and cytoskeletal requirements in erythroblast enucleation. Blood. 2012 Jun 21; 119(25):6118-27. View Signaling and cytoskeletal requirements in erythroblast enucleation. Abstract

  74. Overcoming reprogramming resistance of Fanconi anemia cells. Blood. 2012 Jun 07; 119(23):5449-57. View Overcoming reprogramming resistance of Fanconi anemia cells. Abstract

  75. In vivo enrichment of cytidine deaminase gene-modified hematopoietic cells by prolonged cytosine-arabinoside application. Cytotherapy. 2012 Apr; 14(4):451-60. View In vivo enrichment of cytidine deaminase gene-modified hematopoietic cells by prolonged cytosine-arabinoside application. Abstract

  76. 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 Rac signaling in osteoblastic cells is required for normal bone development but is dispensable for hematopoietic development. Abstract

  77. PTEN negatively regulates engulfment of apoptotic cells by modulating activation of Rac GTPase. J Immunol. 2011 Dec 01; 187(11):5783-94. View PTEN negatively regulates engulfment of apoptotic cells by modulating activation of Rac GTPase. Abstract

  78. 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 Inhibition of Rac GTPase signaling and downstream prosurvival Bcl-2 proteins as combination targeted therapy in MLL-AF9 leukemia. Abstract

  79. Differential niche and Wnt requirements during acute myeloid leukemia progression. Blood. 2011 Sep 08; 118(10):2849-56. View Differential niche and Wnt requirements during acute myeloid leukemia progression. Abstract

  80. 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 Guanine nucleotide exchange factor Vav1 regulates perivascular homing and bone marrow retention of hematopoietic stem and progenitor cells. Abstract

  81. 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 Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting. Abstract

  82. Transatlantic consortium spotlights need for changes in gene therapy trials. Mol Ther. 2010 Nov; 18(11):1892. View Transatlantic consortium spotlights need for changes in gene therapy trials. Abstract

  83. 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 Training program in cancer and blood diseases: Pediatric Hematology/Oncology Fellowship Program, Children's Hospital Boston/Dana-Farber Cancer Institute. Abstract

  84. 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 Gaining the hard yard: pre-clinical evaluation of lentiviral-mediated gene therapy for the treatment of beta-thalassemia. Abstract

  85. 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 The American Society of Pediatric Hematology/Oncology (ASPHO) 2010 Distinguished Career Award Goes to Dr. Sam Lux. Abstract

  86. 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 The Apc(min) mouse has altered hematopoietic stem cell function and provides a model for MPD/MDS. Abstract

  87. Rac GTPases in human diseases. Dis Markers. 2010; 29(3-4):177-87. View Rac GTPases in human diseases. Abstract

  88. Hematology grants workshop. Hematology Am Soc Hematol Educ Program. 2010; 2010:189-90. View Hematology grants workshop. Abstract

  89. 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 Rac1 and Rac2 GTPases are necessary for early erythropoietic expansion in the bone marrow but not in the spleen. Abstract

  90. Correction and apology. Mol Ther. 2009 Oct; 17(10):1660. View Correction and apology. Abstract

  91. National Institutes of Health releases new guidelines on human stem cell research. Mol Ther. 2009 Sep; 17(9):1485-6. View National Institutes of Health releases new guidelines on human stem cell research. Abstract

  92. Gene therapy continues to mature and to face challenges. Mol Ther. 2009 Aug; 17(8):1305-6. View Gene therapy continues to mature and to face challenges. Abstract

  93. Rapid development of pluripotent stem cells as a potential therapeutic modality. Mol Ther. 2009 Jun; 17(6):929-30. View Rapid development of pluripotent stem cells as a potential therapeutic modality. Abstract

  94. Recombinant DNA advisory committee updates recommendations on gene transfer for x-linked severe combined immunodeficiency. Mol Ther. 2009 May; 17(5):751-2. View Recombinant DNA advisory committee updates recommendations on gene transfer for x-linked severe combined immunodeficiency. Abstract

  95. Finding the needle in the hay stack: hematopoietic stem cells in Fanconi anemia. Mutat Res. 2009 Jul 31; 668(1-2):141-9. View Finding the needle in the hay stack: hematopoietic stem cells in Fanconi anemia. Abstract

  96. Progress in genetic therapy for severe combined immunodeficiency associated with adenosine deaminase deficiency. Mol Ther. 2009 Apr; 17(4):577-8. View Progress in genetic therapy for severe combined immunodeficiency associated with adenosine deaminase deficiency. Abstract

  97. Upping the ante: recent advances in direct reprogramming. Mol Ther. 2009 Jun; 17(6):947-53. View Upping the ante: recent advances in direct reprogramming. Abstract

  98. 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 Ectopic HOXB4 overcomes the inhibitory effect of tumor necrosis factor-{alpha} on Fanconi anemia hematopoietic stem and progenitor cells. Abstract

  99. New approaches in the potential treatment of HIV-acquired immunodeficiency disease. Mol Ther. 2009 Feb; 17(2):209-10. View New approaches in the potential treatment of HIV-acquired immunodeficiency disease. Abstract

  100. ESCGT 2008: progress in clinical gene therapy. Mol Ther. 2009 Jan; 17(1):1-2. View ESCGT 2008: progress in clinical gene therapy. Abstract

  101. 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 mTORC1-dependent and -independent regulation of stem cell renewal, differentiation, and mobilization. Abstract

  102. Sleeping beauty vector system moves toward human trials in the United States. Mol Ther. 2008 Sep; 16(9):1515-6. View Sleeping beauty vector system moves toward human trials in the United States. Abstract

  103. Progress reported in two studies of clinical gene transfer into the retina. Mol Ther. 2008 Jul; 16(7):1181. View Progress reported in two studies of clinical gene transfer into the retina. Abstract

  104. 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 Rac GTPase isoforms Rac1 and Rac2 play a redundant and crucial role in T-cell development. Abstract

  105. A "vector drain" in US gene therapy development? Mol Ther. 2008 May; 16(5):801-2. View A "vector drain" in US gene therapy development? Abstract

  106. Rapid lentiviral transduction preserves the engraftment potential of Fanca(-/-) hematopoietic stem cells. Mol Ther. 2008 Jun; 16(6):1154-60. View Rapid lentiviral transduction preserves the engraftment potential of Fanca(-/-) hematopoietic stem cells. Abstract

  107. Foamy virus vectors come of age. Mol Ther. 2008 Apr; 16(4):635-6. View Foamy virus vectors come of age. Abstract

  108. 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 NIH recombinant DNA Advisory Committee continues to ponder adverse event associated with AAV gene therapy trial. Abstract

  109. Rho GTPases and regulation of hematopoietic stem cell localization. Methods Enzymol. 2008; 439:365-93. View Rho GTPases and regulation of hematopoietic stem cell localization. Abstract

  110. 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 Rac1 is essential for intraembryonic hematopoiesis and for the initial seeding of fetal liver with definitive hematopoietic progenitor cells. Abstract

  111. 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 An international conversation on Stem Cell Gene Therapy. 4th Stem Cell Conference on Stem Cell Gene Therapy, Thessaloniki, Greece, 13-17 September 2007. Abstract

  112. RAC reviews serious adverse event associated with AAV therapy trial. Mol Ther. 2007 Dec; 15(12):2053-4. View RAC reviews serious adverse event associated with AAV therapy trial. Abstract

  113. Rac guanosine triphosphatases represent integrating molecular therapeutic targets for BCR-ABL-induced myeloproliferative disease. Cancer Cell. 2007 Nov; 12(5):467-78. View Rac guanosine triphosphatases represent integrating molecular therapeutic targets for BCR-ABL-induced myeloproliferative disease. Abstract

  114. Small pituitary size in children with Fanconi anemia. Pediatr Blood Cancer. 2007 Aug; 49(2):166-70. View Small pituitary size in children with Fanconi anemia. Abstract

  115. NIH decides against continuing NGVLs in their current form. Mol Ther. 2007 Jul; 15(7):1223. View NIH decides against continuing NGVLs in their current form. Abstract

  116. Chemotherapy for myeloid malignancy in children with Fanconi anemia. Pediatr Blood Cancer. 2007 Jun 15; 48(7):668-72. View Chemotherapy for myeloid malignancy in children with Fanconi anemia. Abstract

  117. 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 Live and let die: in vivo selection of gene-modified hematopoietic stem cells via MGMT-mediated chemoprotection. Abstract

  118. Are lentivirus vectors safer? Mol Ther. 2007 Mar; 15(3):439. View Are lentivirus vectors safer? Abstract

  119. 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 Importance of murine study design for testing toxicity of retroviral vectors in support of phase I trials. Abstract

  120. ASGT advises NIH on funding of gene therapy trials. Mol Ther. 2007 Jan; 15(1):1-2. View ASGT advises NIH on funding of gene therapy trials. Abstract

  121. Stem cell collection and gene transfer in Fanconi anemia. Mol Ther. 2007 Jan; 15(1):211-9. View Stem cell collection and gene transfer in Fanconi anemia. Abstract

  122. NIH funding of gene therapy trials. Mol Ther. 2006 Nov; 14(5):607. View NIH funding of gene therapy trials. Abstract

  123. Vector insertion, mutagenesis and transgene toxicity. Mol Ther. 2006 Oct; 14(4):457. View Vector insertion, mutagenesis and transgene toxicity. Abstract

  124. Rac GTPases regulate the morphology and deformability of the erythrocyte cytoskeleton. Blood. 2006 Dec 01; 108(12):3637-45. View Rac GTPases regulate the morphology and deformability of the erythrocyte cytoskeleton. Abstract

  125. A pox on your tumor. Mol Ther. 2006 Sep; 14(3):313. View A pox on your tumor. Abstract

  126. Inhibition of RhoA GTPase activity enhances hematopoietic stem and progenitor cell proliferation and engraftment. Blood. 2006 Sep 15; 108(6):2087-94. View Inhibition of RhoA GTPase activity enhances hematopoietic stem and progenitor cell proliferation and engraftment. Abstract

  127. Gene therapy advances but struggles to interpret safety data in small animal models. Mol Ther. 2006 Jun; 13(6):1027-8. View Gene therapy advances but struggles to interpret safety data in small animal models. Abstract

  128. Adventitious mutations in clinical grade vectors: an issue to consider? Mol Ther. 2006 May; 13(5):831-2. View Adventitious mutations in clinical grade vectors: an issue to consider? Abstract

  129. An emerging consensus on recommendations to facilitate clinical gene transfer. Mol Ther. 2006 Apr; 13(4):637-8. View An emerging consensus on recommendations to facilitate clinical gene transfer. Abstract

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