David A. Williams | Medical Services
Specialties
Programs & Services
Languages
- English
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 | Publications
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
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
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
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
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
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
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
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
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
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
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
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
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
Clonal hematopoiesis in sickle cell disease. J Clin Invest. 2022 02 15; 132(4). View Clonal hematopoiesis in sickle cell disease. 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 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
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
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
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
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
The Changing Face of Adrenoleukodystrophy. Endocr Rev. 2020 08 01; 41(4). View The Changing Face of Adrenoleukodystrophy. 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 Peripheral blood smears of children with multisystem inflammatory syndrome demonstrate prominence of early myeloid forms with morphologic evidence of toxic change. Abstract
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
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
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
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
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
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
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
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
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
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
Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2018 02 01; 378(5):490-491. View Gene Therapy for Cerebral Adrenoleukodystrophy. 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 The gp130 Cytokine Interleukin-11 Regulates Engraftment of Vav1-/- Hematopoietic Stem and Progenitor Cells in Lethally Irradiated Recipients. 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 A key role for Rac and Pak signaling in neutrophil extracellular traps (NETs) formation defines a new potential therapeutic target. Abstract
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
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
Evolving Gene Therapy in Primary Immunodeficiency. Mol Ther. 2017 05 03; 25(5):1132-1141. View Evolving Gene Therapy in Primary Immunodeficiency. Abstract
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
AAV and Insertional Mutagenesis. Mol Ther. 2007 Oct; 15(10):1737. View AAV and Insertional Mutagenesis. Abstract
Of Mouse Models and Men. Mol Ther. 2007 Apr; 15(4):643. View Of Mouse Models and Men. Abstract
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
Unexpected help: mTOR meets lentiviral vectors. Blood. 2014 Aug 07; 124(6):832-3. View Unexpected help: mTOR meets lentiviral vectors. 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 Genetic deletion of the GATA1-regulated protein a-synuclein reduces oxidative stress and nitric oxide synthase levels in mature erythrocytes. Abstract
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
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
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
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
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
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
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
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
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
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
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
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
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
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
Signaling and cytoskeletal requirements in erythroblast enucleation. Blood. 2012 Jun 21; 119(25):6118-27. View Signaling and cytoskeletal requirements in erythroblast enucleation. Abstract
Overcoming reprogramming resistance of Fanconi anemia cells. Blood. 2012 Jun 07; 119(23):5449-57. View Overcoming reprogramming resistance of Fanconi anemia cells. Abstract
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
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
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
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
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
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
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
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
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
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
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
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
Rac GTPases in human diseases. Dis Markers. 2010; 29(3-4):177-87. View Rac GTPases in human diseases. Abstract
Hematology grants workshop. Hematology Am Soc Hematol Educ Program. 2010; 2010:189-90. View Hematology grants workshop. 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 Rac1 and Rac2 GTPases are necessary for early erythropoietic expansion in the bone marrow but not in the spleen. Abstract
Correction and apology. Mol Ther. 2009 Oct; 17(10):1660. View Correction and apology. Abstract
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
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
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
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
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
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
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
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
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
ESCGT 2008: progress in clinical gene therapy. Mol Ther. 2009 Jan; 17(1):1-2. View ESCGT 2008: progress in clinical gene therapy. 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 mTORC1-dependent and -independent regulation of stem cell renewal, differentiation, and mobilization. Abstract
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
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
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
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
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
Foamy virus vectors come of age. Mol Ther. 2008 Apr; 16(4):635-6. View Foamy virus vectors come of age. 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 NIH recombinant DNA Advisory Committee continues to ponder adverse event associated with AAV gene therapy trial. Abstract
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
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
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
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
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
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
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
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
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
Are lentivirus vectors safer? Mol Ther. 2007 Mar; 15(3):439. View Are lentivirus vectors safer? 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 Importance of murine study design for testing toxicity of retroviral vectors in support of phase I trials. Abstract
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
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
NIH funding of gene therapy trials. Mol Ther. 2006 Nov; 14(5):607. View NIH funding of gene therapy trials. Abstract
Vector insertion, mutagenesis and transgene toxicity. Mol Ther. 2006 Oct; 14(4):457. View Vector insertion, mutagenesis and transgene toxicity. Abstract
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
A pox on your tumor. Mol Ther. 2006 Sep; 14(3):313. View A pox on your tumor. Abstract
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
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
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
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
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 Determination of methenamine, methenamine mandelate and methenamine hippurate in pharmaceutical preparations using ion-exchange HPLC. 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 Simultaneous determination of triamcinolone acetonide and oxymetazoline hydrochloride in nasal spray formulations by HPLC. Abstract
New AAV serotypes may broaden the therapeutic pipeline to human gene therapy. Mol Ther. 2006 Jan; 13(1):1-2. View New AAV serotypes may broaden the therapeutic pipeline to human gene therapy. Abstract
FDA guidance document on monitoring delayed adverse events a good first start. Mol Ther. 2005 Dec; 12(6):991-2. View FDA guidance document on monitoring delayed adverse events a good first start. Abstract
Promoting translational research in academic health centers: navigating the "roadmap". Acad Med. 2005 Nov; 80(11):1012-8. View Promoting translational research in academic health centers: navigating the "roadmap". 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 Equal potency of gammaretroviral and lentiviral SIN vectors for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells. 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 Gene therapy in the treatment of Fanconi anemia, a progressive bone marrow failure syndrome. Abstract
A rapid method for retrovirus-mediated identification of complementation groups in Fanconi anemia patients. Mol Ther. 2005 Nov; 12(5):976-84. View A rapid method for retrovirus-mediated identification of complementation groups in Fanconi anemia patients. Abstract
A balanced decision? Regulatory reaction to the "third case". Mol Ther. 2005 Jun; 11(6):819-20. View A balanced decision? Regulatory reaction to the "third case". Abstract
The NIH roadmap: timing is everything. Mol Ther. 2005 Feb; 11(2):173. View The NIH roadmap: timing is everything. 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 Real-Time PCR: an Effective Tool for Measuring Transduction Efficiency in Human Hematopoietic Progenitor Cells. Abstract
Chance or necessity? Insertional mutagenesis in gene therapy and its consequences. Mol Ther. 2004 Jan; 9(1):5-13. View Chance or necessity? Insertional mutagenesis in gene therapy and its consequences. 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 Methylguanine methyltransferase-mediated in vivo selection and chemoprotection of allogeneic stem cells in a large-animal model. Abstract
Medicine. Gene therapy--new challenges ahead. Science. 2003 Oct 17; 302(5644):400-1. View Medicine. Gene therapy--new challenges ahead. 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 A rare complex DNA rearrangement in the murine Steel gene results in exon duplication and a lethal phenotype. Abstract
Side effects of retroviral gene transfer into hematopoietic stem cells. Blood. 2003 Mar 15; 101(6):2099-114. View Side effects of retroviral gene transfer into hematopoietic stem cells. Abstract
Gene Therapy 2000. Hematology Am Soc Hematol Educ Program. 2000; 376-393. View Gene Therapy 2000. Abstract
Mouse model of X-linked chronic granulomatous disease, an inherited defect in phagocyte superoxide production. Nat Genet. 1995 Feb; 9(2):202-9. View Mouse model of X-linked chronic granulomatous disease, an inherited defect in phagocyte superoxide production. Abstract
Novel human oncogene lbc detected by transfection with distinct homology regions to signal transduction products. Oncogene. 1994 Feb; 9(2):621-8. View Novel human oncogene lbc detected by transfection with distinct homology regions to signal transduction products. Abstract
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 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. Abstract
Restriction of neuroblastoma to the prostate gland in transgenic mice. Mol Cell Biol. 1991 Sep; 11(9):4518-27. View Restriction of neuroblastoma to the prostate gland in transgenic mice. Abstract
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 Retroviral gene transfer of human adenosine deaminase in murine hematopoietic cells: effect of selectable marker sequences on long-term expression. Abstract
Gene transfer into murine hematopoietic stem cells and bone marrow stromal cells. Ann N Y Acad Sci. 1990; 612:398-406. View Gene transfer into murine hematopoietic stem cells and bone marrow stromal cells. Abstract
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 Long-term expression of human adenosine deaminase in mice transplanted with retrovirus-infected hematopoietic stem cells. Abstract
Gene therapy of somatic cells: status and prospects. Prog Med Genet. 1988; 7:130-42. View Gene therapy of somatic cells: status and prospects. Abstract
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 Retrovirus-mediated gene transfer of human adenosine deaminase: expression of functional enzyme in murine hematopoietic stem cells in vivo. Abstract
Transfer and expression of human ADA in murine hematopoietic stem cells. Prog Clin Biol Res. 1987; 251:567-80. View Transfer and expression of human ADA in murine hematopoietic stem cells. Abstract
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 Retrovirus-mediated transfer of human adenosine deaminase gene sequences into cells in culture and into murine hematopoietic cells in vivo. Abstract
Somatic gene therapy. Current status and future prospects. J Clin Invest. 1986 Apr; 77(4):1053-6. View Somatic gene therapy. Current status and future prospects. Abstract