Information

Related Research Units

Dana Farber Boston Childrens Cancer and Blood Disorders Center Research
Hematology and Oncology Research
Williams Laboratory Research

Research Overview

Visit the Williams Laboratory

Research in the Williams' laboratory focuses on understanding the biology of the hematopoietic stem cells, including development of gene transfer methods for application in the treatment of severe genetic diseases of the blood system by gene therapy.

The major interest of the Williams laboratory is focused on the biology of hematopoietic stem cells. This work has focused on understanding the interaction of hematopoietic stem cells with the bone marrow and abnormalities of these interactions which are associated with leukemia.

The laboratory is focusing on analysis of the function of members of the Rho GTPase family, specifically Rac and RhoH in blood cell development and function. Rho GTPases are members of the Ras superfamily and act as molecular switches to control multiple cell processes, such as migration, phagocytosis, cell cycle progression, and apoptosis via activation of multiple kinase pathways. Using gene targeted transgenic mice, and a variety of specialized bone marrow culture methods, Dr. Williams' laboratory is defining the essential roles of Rho GTPases in blood cell functions, particularly in response to integrin ligation and activation of chemokine and cytokine receptors. Current work is dissecting the upstream activators and downstream effectors of Rho GTPase in hematopoietic stem cells.

The laboratory has demonstrated that Rac GTPases are key regulators of the engraftment and mobilization functions of hematopoietic stem cells. Increasing focus has been on the dysregulated function of these key molecular switches in leukemia. Recent data from the Williams’ laboratory implicates both Rac and Rho in acute and chronic leukemias and current efforts include development of new inhibitors of these molecular targets and validation studies of these molecules in leukemia model systems. In addition, this basic work has helped to define the molecular abnormalities in two rare immunodeficiency diseases, Leukocyte Adhesion Deficiency Type IV (due to RAC2 mutations) and Epidermodysplasia Verruciformis (due to mutations in RHOH).

Much of the basic information derived from these studies is also being applied to improve the methods of gene transfer into hematopoietic stem cells using retrovirus and lentivirus vectors.
Dr. Williams is serving as sponsor investigator for a number of human gene therapy trials at CHB, including in SCID-X1, Wiskott-Aldrich Disease, Childhood Cerebral Adrenoleukodystropy and a trial in sickle cell disease targeting the transcription factor BCL11a.

Major goals of research:

  • To further understand the role Rho GTPases as key regulatory switches that control stem cell adhesion, migration and survival/proliferation.
  • Continue to develop and advance the use viral vectors for gene transfer into hematopoietic stem cells with the purpose of advancing clinical gene therapy trials.
  • To advance the translation of basic research into novel therapeutic applications.

Spotlights:

  • A phase 1/2 clinical trial in Blood reports encouraging results with gene therapy in patients with Wiskott-Aldrich syndrome, using a self-inactivating lentiviral vector. Click here to read  more
     
  • A study in Nature Communications confirms that gene therapy targeting BCL11A for sickle-cell disease boosted patients’ non-sickling fetal hemoglobin while reducing adult hemoglobin. Click here to read more

Research Background

Dr. Williams has won numerous prestigious awards for his research, including: the Dameshek Award and the Frank Oski Award of the American Society of Hematology for research in hematology; the Donald Metcalf Award from the International Society for Hematology and Stem Cells for contributions to the field; the E. Mead Johnson Award of the Society for Pediatric Research for research in pediatrics and most recently the Outstanding Achievement Award of the American Society of Gene and Cell Therapy (2011). He was an Investigator of the Howard Hughes Medical Institute for 16 years and is a member of the National Academy of Sciences Institute of Medicine. He has been continuously funded by the National Institutes of Health since 1986. Dr. Williams has multiple patents, several of which have been licensed to pharmaceutical and biotechnology.

Dr. Williams' Clinical Page

 

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

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

Publications

  1. Myelodysplasia after Lentiviral Gene Therapy. Reply. N Engl J Med. 2024 Dec 19; 391(24):2384. View Abstract
  2. Gene editing without ex vivo culture evades genotoxicity in human hematopoietic stem cells. Cell Stem Cell. 2024 Dec 11. View Abstract
  3. 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 Abstract
  4. Engineered packaging cell line for the enhanced production of baboon-enveloped retroviral vectors. Mol Ther Nucleic Acids. 2024 Dec 10; 35(4):102389. View Abstract
  5. Regulated GATA1 expression as a universal gene therapy for Diamond-Blackfan anemia. Cell Stem Cell. 2025 Jan 02; 32(1):38-52.e6. View Abstract
  6. Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2024 Oct 10; 391(14):1287-1301. View Abstract
  7. Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2024 Oct 10; 391(14):1302-1312. View Abstract
  8. 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 Abstract
  9. Clonal selection of hematopoietic stem cells after gene therapy for sickle cell disease. Nat Med. 2023 Dec; 29(12):3175-3183. View Abstract
  10. Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome. Blood. 2023 10 12; 142(15):1281-1296. View Abstract
  11. 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 Abstract
  12. 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 Abstract
  13. 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 Abstract
  14. The long road traveled in hematopoietic stem cell gene therapy. Mol Ther. 2022 10 05; 30(10):3097-3099. View Abstract
  15. 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 Abstract
  16. 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 Abstract
  17. Clonal hematopoiesis in sickle cell disease. J Clin Invest. 2022 02 15; 132(4). View Abstract
  18. 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 Abstract
  19. Investigational curative gene therapy approaches to sickle cell disease. Blood Adv. 2021 12 14; 5(23):5452. View Abstract
  20. 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
  21. Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease. N Engl J Med. 2021 01 21; 384(3):205-215. View Abstract
  22. 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
  23. The Changing Face of Adrenoleukodystrophy. Endocr Rev. 2020 08 01; 41(4). View Abstract
  24. 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
  25. Children's rare disease cohorts: an integrative research and clinical genomics initiative. NPJ Genom Med. 2020; 5:29. View Abstract
  26. Children's rare disease cohorts: an integrative research and clinical genomics initiative. NPJ Genom Med. 2020 Jul 06; 5(1):29. View Abstract
  27. Current and future gene therapies for hemoglobinopathies. Curr Opin Hematol. 2020 05; 27(3):149-154. View Abstract
  28. Infantile Myelofibrosis and Myeloproliferation with CDC42 Dysfunction. J Clin Immunol. 2020 05; 40(4):554-566. View Abstract
  29. 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
  30. Highly efficient therapeutic gene editing of human hematopoietic stem cells. Nat Med. 2019 May; 25(5):776-783. View Abstract
  31. 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
  32. 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
  33. The pseudokinase MLKL activates PAD4-dependent NET formation in necroptotic neutrophils. Sci Signal. 2018 09 04; 11(546). View Abstract
  34. 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
  35. Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2018 02 01; 378(5):490-491. View Abstract
  36. 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
  37. 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
  38. Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2017 10 26; 377(17):1630-1638. View Abstract
  39. The 2017 ASPHO distinguished career award goes to Holcombe E. Grier, MD. Pediatr Blood Cancer. 2017 06; 64 Suppl 1. View Abstract
  40. Evolving Gene Therapy in Primary Immunodeficiency. Mol Ther. 2017 05 03; 25(5):1132-1141. View Abstract
  41. Rapid Lentiviral Transduction Preserves the Engraftment Potential of Fanca-/- Hematopoietic Stem Cells. Mol Ther. 2008 Jun; 16(6):1154-1160. View Abstract
  42. AAV and Insertional Mutagenesis. Mol Ther. 2007 Oct; 15(10):1737. View Abstract
  43. Of Mouse Models and Men. Mol Ther. 2007 Apr; 15(4):643. View Abstract
  44. Molecular Therapy Moves on to a New Editor-in-Chief. Mol Ther. 2009 Dec; 17(12):1989-1990. View Abstract
  45. Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype. J Clin Invest. 2016 10 03; 126(10):3868-3878. View Abstract
  46. Assessment of physical function and participation in chronic pain clinical trials: IMMPACT/OMERACT recommendations. Pain. 2016 Sep; 157(9):1836-1850. View Abstract
  47. 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
  48. Two Phase 3 Trials of Adalimumab for Hidradenitis Suppurativa. N Engl J Med. 2016 Aug 04; 375(5):422-34. View Abstract
  49. Mathematical modeling of erythrocyte chimerism informs genetic intervention strategies for sickle cell disease. Am J Hematol. 2016 09; 91(9):931-7. View Abstract
  50. 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
  51. Mechanism of Drug-Drug Interactions Between Warfarin and Statins. J Pharm Sci. 2016 06; 105(6):1976-1986. View Abstract
  52. 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
  53. 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
  54. 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
  55. 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
  56. Flow cytometric assay for direct quantification of neutrophil extracellular traps in blood samples. Am J Hematol. 2015 Dec; 90(12):1155-8. View Abstract
  57. miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction. Mol Ther. 2015 Sep; 23(9):1465-74. View Abstract
  58. 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
  59. A modified ?-retrovirus vector for X-linked severe combined immunodeficiency. N Engl J Med. 2014 Oct 09; 371(15):1407-17. View Abstract
  60. Unexpected help: mTOR meets lentiviral vectors. Blood. 2014 Aug 07; 124(6):832-3. View Abstract
  61. 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
  62. Charting a clear path: the ASGCT Standardized Pathways Conference. Mol Ther. 2014 Jul; 22(7):1235-1238. View Abstract
  63. 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
  64. 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
  65. Retroviral transduction of murine and human hematopoietic progenitors and stem cells. Methods Mol Biol. 2014; 1185:287-309. View Abstract
  66. 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
  67. 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
  68. Oxandrolone for the treatment of bone marrow failure in Fanconi anemia. Pediatr Blood Cancer. 2014 Jan; 61(1):11-9. View Abstract
  69. Broadening the indications for hematopoietic stem cell genetic therapies. Cell Stem Cell. 2013 Sep 05; 13(3):263-4. View Abstract
  70. 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
  71. 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
  72. Enough is indeed enough: ACGME required changes in pediatric training. Pediatr Blood Cancer. 2012 Dec 15; 59(7):1158-9. View Abstract
  73. 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
  74. CTC1 Mutations in a patient with dyskeratosis congenita. Pediatr Blood Cancer. 2012 Aug; 59(2):311-4. View Abstract
  75. 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
  76. Signaling and cytoskeletal requirements in erythroblast enucleation. Blood. 2012 Jun 21; 119(25):6118-27. View Abstract
  77. Overcoming reprogramming resistance of Fanconi anemia cells. Blood. 2012 Jun 07; 119(23):5449-57. View Abstract
  78. In vivo enrichment of cytidine deaminase gene-modified hematopoietic cells by prolonged cytosine-arabinoside application. Cytotherapy. 2012 Apr; 14(4):451-60. View Abstract
  79. 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
  80. PTEN negatively regulates engulfment of apoptotic cells by modulating activation of Rac GTPase. J Immunol. 2011 Dec 01; 187(11):5783-94. View Abstract
  81. 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
  82. Differential niche and Wnt requirements during acute myeloid leukemia progression. Blood. 2011 Sep 08; 118(10):2849-56. View Abstract
  83. 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
  84. 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
  85. Transatlantic consortium spotlights need for changes in gene therapy trials. Mol Ther. 2010 Nov; 18(11):1892. View Abstract
  86. 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
  87. 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
  88. 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
  89. 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
  90. Rac GTPases in human diseases. Dis Markers. 2010; 29(3-4):177-87. View Abstract
  91. Hematology grants workshop. Hematology Am Soc Hematol Educ Program. 2010; 2010:189-90. View Abstract
  92. 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
  93. Correction and apology. Mol Ther. 2009 Oct; 17(10):1660. View Abstract
  94. National Institutes of Health releases new guidelines on human stem cell research. Mol Ther. 2009 Sep; 17(9):1485-6. View Abstract
  95. Gene therapy continues to mature and to face challenges. Mol Ther. 2009 Aug; 17(8):1305-6. View Abstract
  96. Rapid development of pluripotent stem cells as a potential therapeutic modality. Mol Ther. 2009 Jun; 17(6):929-30. View Abstract
  97. 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
  98. 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
  99. Progress in genetic therapy for severe combined immunodeficiency associated with adenosine deaminase deficiency. Mol Ther. 2009 Apr; 17(4):577-8. View Abstract
  100. Upping the ante: recent advances in direct reprogramming. Mol Ther. 2009 Jun; 17(6):947-53. View Abstract
  101. 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
  102. New approaches in the potential treatment of HIV-acquired immunodeficiency disease. Mol Ther. 2009 Feb; 17(2):209-10. View Abstract
  103. ESCGT 2008: progress in clinical gene therapy. Mol Ther. 2009 Jan; 17(1):1-2. View Abstract
  104. 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
  105. Sleeping beauty vector system moves toward human trials in the United States. Mol Ther. 2008 Sep; 16(9):1515-6. View Abstract
  106. Progress reported in two studies of clinical gene transfer into the retina. Mol Ther. 2008 Jul; 16(7):1181. View Abstract
  107. 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
  108. A "vector drain" in US gene therapy development? Mol Ther. 2008 May; 16(5):801-2. View Abstract
  109. Rapid lentiviral transduction preserves the engraftment potential of Fanca(-/-) hematopoietic stem cells. Mol Ther. 2008 Jun; 16(6):1154-60. View Abstract
  110. Foamy virus vectors come of age. Mol Ther. 2008 Apr; 16(4):635-6. View Abstract
  111. 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
  112. Rho GTPases and regulation of hematopoietic stem cell localization. Methods Enzymol. 2008; 439:365-93. View Abstract
  113. 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
  114. 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
  115. RAC reviews serious adverse event associated with AAV therapy trial. Mol Ther. 2007 Dec; 15(12):2053-4. View Abstract
  116. Rac guanosine triphosphatases represent integrating molecular therapeutic targets for BCR-ABL-induced myeloproliferative disease. Cancer Cell. 2007 Nov; 12(5):467-78. View Abstract
  117. Small pituitary size in children with Fanconi anemia. Pediatr Blood Cancer. 2007 Aug; 49(2):166-70. View Abstract
  118. NIH decides against continuing NGVLs in their current form. Mol Ther. 2007 Jul; 15(7):1223. View Abstract
  119. Chemotherapy for myeloid malignancy in children with Fanconi anemia. Pediatr Blood Cancer. 2007 Jun 15; 48(7):668-72. View Abstract
  120. 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
  121. Are lentivirus vectors safer? Mol Ther. 2007 Mar; 15(3):439. View Abstract
  122. 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
  123. ASGT advises NIH on funding of gene therapy trials. Mol Ther. 2007 Jan; 15(1):1-2. View Abstract
  124. Stem cell collection and gene transfer in Fanconi anemia. Mol Ther. 2007 Jan; 15(1):211-9. View Abstract
  125. NIH funding of gene therapy trials. Mol Ther. 2006 Nov; 14(5):607. View Abstract
  126. Vector insertion, mutagenesis and transgene toxicity. Mol Ther. 2006 Oct; 14(4):457. View Abstract
  127. Rac GTPases regulate the morphology and deformability of the erythrocyte cytoskeleton. Blood. 2006 Dec 01; 108(12):3637-45. View Abstract
  128. A pox on your tumor. Mol Ther. 2006 Sep; 14(3):313. View Abstract
  129. Inhibition of RhoA GTPase activity enhances hematopoietic stem and progenitor cell proliferation and engraftment. Blood. 2006 Sep 15; 108(6):2087-94. View Abstract
  130. Gene therapy advances but struggles to interpret safety data in small animal models. Mol Ther. 2006 Jun; 13(6):1027-8. View Abstract
  131. Adventitious mutations in clinical grade vectors: an issue to consider? Mol Ther. 2006 May; 13(5):831-2. View Abstract
  132. An emerging consensus on recommendations to facilitate clinical gene transfer. Mol Ther. 2006 Apr; 13(4):637-8. View Abstract
  133. 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
  134. 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
  135. New AAV serotypes may broaden the therapeutic pipeline to human gene therapy. Mol Ther. 2006 Jan; 13(1):1-2. View Abstract
  136. FDA guidance document on monitoring delayed adverse events a good first start. Mol Ther. 2005 Dec; 12(6):991-2. View Abstract
  137. Promoting translational research in academic health centers: navigating the "roadmap". Acad Med. 2005 Nov; 80(11):1012-8. View Abstract
  138. 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
  139. 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
  140. A rapid method for retrovirus-mediated identification of complementation groups in Fanconi anemia patients. Mol Ther. 2005 Nov; 12(5):976-84. View Abstract
  141. A balanced decision? Regulatory reaction to the "third case". Mol Ther. 2005 Jun; 11(6):819-20. View Abstract
  142. The NIH roadmap: timing is everything. Mol Ther. 2005 Feb; 11(2):173. View Abstract
  143. 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
  144. Chance or necessity? Insertional mutagenesis in gene therapy and its consequences. Mol Ther. 2004 Jan; 9(1):5-13. View Abstract
  145. 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
  146. Medicine. Gene therapy--new challenges ahead. Science. 2003 Oct 17; 302(5644):400-1. View Abstract
  147. 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
  148. Side effects of retroviral gene transfer into hematopoietic stem cells. Blood. 2003 Mar 15; 101(6):2099-114. View Abstract
  149. Gene Therapy 2000. Hematology Am Soc Hematol Educ Program. 2000; 376-393. View Abstract
  150. 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
  151. Novel human oncogene lbc detected by transfection with distinct homology regions to signal transduction products. Oncogene. 1994 Feb; 9(2):621-8. View Abstract
  152. 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
  153. Restriction of neuroblastoma to the prostate gland in transgenic mice. Mol Cell Biol. 1991 Sep; 11(9):4518-27. View Abstract
  154. 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
  155. Gene transfer into murine hematopoietic stem cells and bone marrow stromal cells. Ann N Y Acad Sci. 1990; 612:398-406. View Abstract
  156. 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
  157. Gene therapy of somatic cells: status and prospects. Prog Med Genet. 1988; 7:130-42. View Abstract
  158. 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
  159. Transfer and expression of human ADA in murine hematopoietic stem cells. Prog Clin Biol Res. 1987; 251:567-80. View Abstract
  160. 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
  161. Somatic gene therapy. Current status and future prospects. J Clin Invest. 1986 Apr; 77(4):1053-6. View Abstract

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