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.
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
Myelodysplasia after Lentiviral Gene Therapy. Reply. N Engl J Med. 2024 Dec 19; 391(24):2384. View Abstract
Gene editing without ex vivo culture evades genotoxicity in human hematopoietic stem cells. Cell Stem Cell. 2024 Dec 11. View Abstract
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
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
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
Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2024 Oct 10; 391(14):1287-1301. View Abstract
Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2024 Oct 10; 391(14):1302-1312. View Abstract
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
Clonal selection of hematopoietic stem cells after gene therapy for sickle cell disease. Nat Med. 2023 Dec; 29(12):3175-3183. View Abstract
Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome. Blood. 2023 10 12; 142(15):1281-1296. View Abstract
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
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
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
The long road traveled in hematopoietic stem cell gene therapy. Mol Ther. 2022 10 05; 30(10):3097-3099. View Abstract
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
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
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
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
Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease. N Engl J Med. 2021 01 21; 384(3):205-215. View Abstract
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
The Changing Face of Adrenoleukodystrophy. Endocr Rev. 2020 08 01; 41(4). View Abstract
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
Children's rare disease cohorts: an integrative research and clinical genomics initiative. NPJ Genom Med. 2020; 5:29. View Abstract
Children's rare disease cohorts: an integrative research and clinical genomics initiative. NPJ Genom Med. 2020 Jul 06; 5(1):29. View Abstract
Current and future gene therapies for hemoglobinopathies. Curr Opin Hematol. 2020 05; 27(3):149-154. View Abstract
Infantile Myelofibrosis and Myeloproliferation with CDC42 Dysfunction. J Clin Immunol. 2020 05; 40(4):554-566. View Abstract
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
Highly efficient therapeutic gene editing of human hematopoietic stem cells. Nat Med. 2019 May; 25(5):776-783. View Abstract
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
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
The pseudokinase MLKL activates PAD4-dependent NET formation in necroptotic neutrophils. Sci Signal. 2018 09 04; 11(546). View Abstract
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
Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2018 02 01; 378(5):490-491. View Abstract
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
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
Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2017 10 26; 377(17):1630-1638. View Abstract
The 2017 ASPHO distinguished career award goes to Holcombe E. Grier, MD. Pediatr Blood Cancer. 2017 06; 64 Suppl 1. View Abstract
Assessment of physical function and participation in chronic pain clinical trials: IMMPACT/OMERACT recommendations. Pain. 2016 Sep; 157(9):1836-1850. View Abstract
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
Two Phase 3 Trials of Adalimumab for Hidradenitis Suppurativa. N Engl J Med. 2016 Aug 04; 375(5):422-34. View Abstract
Mathematical modeling of erythrocyte chimerism informs genetic intervention strategies for sickle cell disease. Am J Hematol. 2016 09; 91(9):931-7. View Abstract
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
Mechanism of Drug-Drug Interactions Between Warfarin and Statins. J Pharm Sci. 2016 06; 105(6):1976-1986. View Abstract
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
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
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
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
Flow cytometric assay for direct quantification of neutrophil extracellular traps in blood samples. Am J Hematol. 2015 Dec; 90(12):1155-8. View Abstract
miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction. Mol Ther. 2015 Sep; 23(9):1465-74. View Abstract
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
A modified ?-retrovirus vector for X-linked severe combined immunodeficiency. N Engl J Med. 2014 Oct 09; 371(15):1407-17. View Abstract
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
Charting a clear path: the ASGCT Standardized Pathways Conference. Mol Ther. 2014 Jul; 22(7):1235-1238. View Abstract
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
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
Retroviral transduction of murine and human hematopoietic progenitors and stem cells. Methods Mol Biol. 2014; 1185:287-309. View Abstract
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
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
Oxandrolone for the treatment of bone marrow failure in Fanconi anemia. Pediatr Blood Cancer. 2014 Jan; 61(1):11-9. View Abstract
Broadening the indications for hematopoietic stem cell genetic therapies. Cell Stem Cell. 2013 Sep 05; 13(3):263-4. View Abstract
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
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
Enough is indeed enough: ACGME required changes in pediatric training. Pediatr Blood Cancer. 2012 Dec 15; 59(7):1158-9. View Abstract
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
CTC1 Mutations in a patient with dyskeratosis congenita. Pediatr Blood Cancer. 2012 Aug; 59(2):311-4. View Abstract
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
Signaling and cytoskeletal requirements in erythroblast enucleation. Blood. 2012 Jun 21; 119(25):6118-27. View Abstract
Overcoming reprogramming resistance of Fanconi anemia cells. Blood. 2012 Jun 07; 119(23):5449-57. View Abstract
In vivo enrichment of cytidine deaminase gene-modified hematopoietic cells by prolonged cytosine-arabinoside application. Cytotherapy. 2012 Apr; 14(4):451-60. View Abstract
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
PTEN negatively regulates engulfment of apoptotic cells by modulating activation of Rac GTPase. J Immunol. 2011 Dec 01; 187(11):5783-94. View Abstract
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
Differential niche and Wnt requirements during acute myeloid leukemia progression. Blood. 2011 Sep 08; 118(10):2849-56. View Abstract
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
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
Transatlantic consortium spotlights need for changes in gene therapy trials. Mol Ther. 2010 Nov; 18(11):1892. View Abstract
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
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
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
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
Rac GTPases in human diseases. Dis Markers. 2010; 29(3-4):177-87. View Abstract
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
Correction and apology. Mol Ther. 2009 Oct; 17(10):1660. View Abstract
National Institutes of Health releases new guidelines on human stem cell research. Mol Ther. 2009 Sep; 17(9):1485-6. View Abstract
Gene therapy continues to mature and to face challenges. Mol Ther. 2009 Aug; 17(8):1305-6. View Abstract
Rapid development of pluripotent stem cells as a potential therapeutic modality. Mol Ther. 2009 Jun; 17(6):929-30. View Abstract
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
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
Progress in genetic therapy for severe combined immunodeficiency associated with adenosine deaminase deficiency. Mol Ther. 2009 Apr; 17(4):577-8. View Abstract
Upping the ante: recent advances in direct reprogramming. Mol Ther. 2009 Jun; 17(6):947-53. View Abstract
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
New approaches in the potential treatment of HIV-acquired immunodeficiency disease. Mol Ther. 2009 Feb; 17(2):209-10. View Abstract
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
Sleeping beauty vector system moves toward human trials in the United States. Mol Ther. 2008 Sep; 16(9):1515-6. View Abstract
Progress reported in two studies of clinical gene transfer into the retina. Mol Ther. 2008 Jul; 16(7):1181. View Abstract
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
A "vector drain" in US gene therapy development? Mol Ther. 2008 May; 16(5):801-2. View Abstract
Rapid lentiviral transduction preserves the engraftment potential of Fanca(-/-) hematopoietic stem cells. Mol Ther. 2008 Jun; 16(6):1154-60. View Abstract
Foamy virus vectors come of age. Mol Ther. 2008 Apr; 16(4):635-6. View Abstract
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
Rho GTPases and regulation of hematopoietic stem cell localization. Methods Enzymol. 2008; 439:365-93. View Abstract
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
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
Rac guanosine triphosphatases represent integrating molecular therapeutic targets for BCR-ABL-induced myeloproliferative disease. Cancer Cell. 2007 Nov; 12(5):467-78. View Abstract
Small pituitary size in children with Fanconi anemia. Pediatr Blood Cancer. 2007 Aug; 49(2):166-70. View Abstract
NIH decides against continuing NGVLs in their current form. Mol Ther. 2007 Jul; 15(7):1223. View Abstract
Chemotherapy for myeloid malignancy in children with Fanconi anemia. Pediatr Blood Cancer. 2007 Jun 15; 48(7):668-72. View Abstract
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
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
ASGT advises NIH on funding of gene therapy trials. Mol Ther. 2007 Jan; 15(1):1-2. View Abstract
Stem cell collection and gene transfer in Fanconi anemia. Mol Ther. 2007 Jan; 15(1):211-9. View Abstract
Rac GTPases regulate the morphology and deformability of the erythrocyte cytoskeleton. Blood. 2006 Dec 01; 108(12):3637-45. View Abstract
A pox on your tumor. Mol Ther. 2006 Sep; 14(3):313. View Abstract
Inhibition of RhoA GTPase activity enhances hematopoietic stem and progenitor cell proliferation and engraftment. Blood. 2006 Sep 15; 108(6):2087-94. View Abstract
Gene therapy advances but struggles to interpret safety data in small animal models. Mol Ther. 2006 Jun; 13(6):1027-8. View Abstract
Adventitious mutations in clinical grade vectors: an issue to consider? Mol Ther. 2006 May; 13(5):831-2. View Abstract
An emerging consensus on recommendations to facilitate clinical gene transfer. Mol Ther. 2006 Apr; 13(4):637-8. View Abstract
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
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
New AAV serotypes may broaden the therapeutic pipeline to human gene therapy. Mol Ther. 2006 Jan; 13(1):1-2. View Abstract
FDA guidance document on monitoring delayed adverse events a good first start. Mol Ther. 2005 Dec; 12(6):991-2. View Abstract
Promoting translational research in academic health centers: navigating the "roadmap". Acad Med. 2005 Nov; 80(11):1012-8. View Abstract
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
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
A rapid method for retrovirus-mediated identification of complementation groups in Fanconi anemia patients. Mol Ther. 2005 Nov; 12(5):976-84. View Abstract
A balanced decision? Regulatory reaction to the "third case". Mol Ther. 2005 Jun; 11(6):819-20. View Abstract
The NIH roadmap: timing is everything. Mol Ther. 2005 Feb; 11(2):173. View Abstract
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
Chance or necessity? Insertional mutagenesis in gene therapy and its consequences. Mol Ther. 2004 Jan; 9(1):5-13. View Abstract
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
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
Side effects of retroviral gene transfer into hematopoietic stem cells. Blood. 2003 Mar 15; 101(6):2099-114. View Abstract
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