David A. Williams

Eltrombopag in combination with immunosuppressive therapy in pediatric severe aplastic anemia: Phase 2 ESCALATE trial. Blood Adv. 2025 May 02. View Eltrombopag in combination with immunosuppressive therapy in pediatric severe aplastic anemia: Phase 2 ESCALATE trial. Abstract

Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy. Mol Ther. 2025 May 07; 33(5):2141-2153. View Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy. Abstract

Myelodysplasia after Lentiviral Gene Therapy. Reply. N Engl J Med. 2024 Dec 19; 391(24):2384. View Myelodysplasia after Lentiviral Gene Therapy. Reply. Abstract

Gene editing without ex vivo culture evades genotoxicity in human hematopoietic stem cells. Cell Stem Cell. 2025 Feb 06; 32(2):191-208.e11. View Gene editing without ex vivo culture evades genotoxicity in human hematopoietic stem cells. 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 UM171 enhances fitness and engraftment of gene-modified hematopoietic stem cells from patients with sickle cell disease. 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 Engineered packaging cell line for the enhanced production of baboon-enveloped retroviral vectors. 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 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 10 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 10 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

Gene editing without ex vivo culture evades genotoxicity in human hematopoietic stem cells. bioRxiv. 2023 May 27. View Gene editing without ex vivo culture evades genotoxicity in human hematopoietic stem cells. 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

Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop. Hum Gene Ther. 2023 02; 34(3-4):83-89. View Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop. Abstract

Secondary Neoplasms After Hematopoietic Cell Transplant for Sickle Cell Disease. J Clin Oncol. 2023 04 20; 41(12):2227-2237. View Secondary Neoplasms After Hematopoietic Cell Transplant for Sickle Cell Disease. 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

Restored Macrophage Function Ameliorates Disease Pathophysiology in a Mouse Model for IL10 Receptor-deficient Very Early Onset Inflammatory Bowel Disease. J Crohns Colitis. 2021 Sep 25; 15(9):1588-1595. View Restored Macrophage Function Ameliorates Disease Pathophysiology in a Mouse Model for IL10 Receptor-deficient Very Early Onset Inflammatory Bowel Disease. Abstract

Evidence generation and reproducibility in cell and gene therapy research: A call to action. Mol Ther Methods Clin Dev. 2021 Sep 10; 22:11-14. View Evidence generation and reproducibility in cell and gene therapy research: A call to action. 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

Lentiviral gene therapy for X-linked chronic granulomatous disease. Nat Med. 2020 02; 26(2):200-206. View Lentiviral gene therapy for X-linked chronic granulomatous disease. 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

T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency. Genome Med. 2018 09 28; 10(1):70. View T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency. 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

Self-Guided Online Cognitive Behavioral Strategies for Chemotherapy-Induced Peripheral Neuropathy: A Multicenter, Pilot, Randomized, Wait-List Controlled Trial. J Pain. 2018 04; 19(4):382-394. View Self-Guided Online Cognitive Behavioral Strategies for Chemotherapy-Induced Peripheral Neuropathy: A Multicenter, Pilot, Randomized, Wait-List Controlled Trial. 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

Enhancement of mouse hematopoietic stem/progenitor cell function via transient gene delivery using integration-deficient lentiviral vectors. Exp Hematol. 2018 01; 57:21-29. View Enhancement of mouse hematopoietic stem/progenitor cell function via transient gene delivery using integration-deficient lentiviral vectors. 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

Curing genetic disease with gene therapy. Trans Am Clin Climatol Assoc. 2014; 125:122-8; discussion 128-9. View Curing genetic disease with gene therapy. 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

Out of harm's way. Nat Biotechnol. 2011 Jan; 29(1):41-2. View Out of harm's way. 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

Reciprocal relationship between O6-methylguanine-DNA methyltransferase P140K expression level and chemoprotection of hematopoietic stem cells. Cancer Res. 2008 Aug 01; 68(15):6171-80. View Reciprocal relationship between O6-methylguanine-DNA methyltransferase P140K expression level and chemoprotection of hematopoietic stem cells. 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

Lentiviral vectors pseudotyped with murine ecotropic envelope: increased biosafety and convenience in preclinical research. Exp Hematol. 2006 May; 34(5):588-92. View Lentiviral vectors pseudotyped with murine ecotropic envelope: increased biosafety and convenience in preclinical research. 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

Comparison of three retroviral vector systems for transduction of nonobese diabetic/severe combined immunodeficiency mice repopulating human CD34+ cord blood cells. Hum Gene Ther. 2003 Apr 10; 14(6):509-19. View Comparison of three retroviral vector systems for transduction of nonobese diabetic/severe combined immunodeficiency mice repopulating human CD34+ cord blood cells. 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

Hematoprotection and enrichment of transduced cells in vivo after gene transfer of MGMT(P140K) into hematopoietic stem cells. Cancer Gene Ther. 2002 Sep; 9(9):737-46. View Hematoprotection and enrichment of transduced cells in vivo after gene transfer of MGMT(P140K) 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