David A. Williams

Publications

1. PCORnet®: An Infrastructure Supporting Innovation in Clinical Study Design. Med Care. 2026 Feb 01; 64(2S Suppl 3):S178-S184. View Abstract
2. Urologic chronic pelvic pain syndrome 3-year symptom trajectories: the Multidisciplinary Approach to the Study of Chronic Pelvic Pain (MAPP) Symptom Patterns Study. BJU Int. 2026 Feb; 137(2):312-322. View Abstract
3. Baseline characteristics of participants in the Biomarkers for Evaluating Spine Treatments clinical trial: a sequential multiple assignment randomized trial for chronic low back pain†. Pain Med. 2025 Nov 01; 26(11):758-772. View Abstract
4. Long COVID associated with SARS-CoV-2 reinfection among children and adolescents in the omicron era (RECOVER-EHR): a retrospective cohort study. Lancet Infect Dis. 2026 Feb; 26(2):127-138. View Abstract
5. Consanguinity and treatment strategy determine seizure outcome and mortality in infantile epileptic spasms syndrome in Azerbaijan. Seizure. 2025 Nov; 132:125-132. View Abstract
6. The design and rationale of the Biomarkers for Evaluating Spine Treatments trial: a sequential multiple assignment randomized trial. Pain Med. 2025 Sep 01; 26(9):538-553. View Abstract
7. Respiratory and Other Infections Following COVID. Pediatrics. 2025 Sep 01; 156(3). View Abstract
8. Eltrombopag in combination with immunosuppressive therapy in pediatric severe aplastic anemia: phase 2 ESCALATE trial. Blood Adv. 2025 Aug 12; 9(15):3728-3738. View Abstract
9. Risk of neuropsychiatric and related conditions associated with SARS-CoV-2 infection: a difference-in-differences analysis. Nat Commun. 2025 Jul 24; 16(1):6829. View Abstract
10. Regression of Monosomy 7 Clone in Patient With RECQL4-Associated Syndrome. Am J Hematol. 2025 Sep; 100(9):1631-1642. View Abstract
11. Towards precision medicine in clinical trials for the treatment of urologic chronic pelvic pain syndrome: lessons from the MAPP Research Network. Nat Rev Urol. 2025 Sep; 22(9):632-642. View Abstract
12. Cardiovascular post-acute sequelae of SARS-CoV-2 in children and adolescents: cohort study using electronic health records. Nat Commun. 2025 Apr 11; 16(1):3445. View Abstract
13. Pediatric Long COVID Subphenotypes: An EHR-based study from the RECOVER program. PLOS Digit Health. 2025 Apr; 4(4):e0000747. View Abstract
14. High-impact Chronic Pain in a Cohort of Urologic Chronic Pelvic Pain Syndrome Patients: A Retrospective MAPP Research Network Study. Clin J Pain. 2025 Apr 01; 41(4). View Abstract
15. Kidney Function Following COVID-19 in Children and Adolescents. JAMA Netw Open. 2025 04 01; 8(4):e254129. View Abstract
16. Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy. Mol Ther. 2025 May 07; 33(5):2141-2153. View Abstract
17. Reinfection with SARS-CoV-2 in the Omicron Era is Associated with Increased Risk of Post-Acute Sequelae of SARS-CoV-2 Infection: A RECOVER-EHR Cohort Study. medRxiv. 2025 Mar 30. View Abstract
18. Scientific Advancements in Gene Therapies: Opportunities for Global Regulatory Convergence. Biomedicines. 2025 Mar 20; 13(3). View Abstract
19. Widespread pain phenotypes impact treatment efficacy results in randomized clinical trials for interstitial cystitis/bladder pain syndrome: a Multidisciplinary Approach to the Study of Chronic Pelvic Pain network study. Pain. 2025 May 01; 166(5):1179-1190. View Abstract
20. Vaccine Effectiveness Among 5- to 17-year-old Individuals with Prior SARS-CoV-2 Infection: An EHR-Based Target Trial Emulation Study from the RECOVER Project. medRxiv. 2025 Feb 08. View Abstract
21. Racial/ethnic differences in post-acute sequelae of SARS-CoV-2 in children and adolescents in the United States. Nat Commun. 2025 Jan 21; 16(1):878. View Abstract
22. Myelodysplasia after Lentiviral Gene Therapy. Reply. N Engl J Med. 2024 Dec 19; 391(24):2384. View Abstract
23. 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 Abstract
24. 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
25. 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
26. 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
27. Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2024 10 10; 391(14):1287-1301. View Abstract
28. Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2024 10 10; 391(14):1302-1312. View Abstract
29. Body Mass Index and Postacute Sequelae of SARS-CoV-2 Infection in Children and Young Adults. JAMA Netw Open. 2024 10 01; 7(10):e2441970. View Abstract
30. Pediatric Long COVID Subphenotypes: An EHR-based study from the RECOVER program. medRxiv. 2024 Sep 18. View Abstract
31. Pediatric Gastrointestinal Outcomes During the Post-Acute Phase of COVID-19: Findings from RECOVER Initiative from 29 Hospitals in the US. medRxiv. 2024 Jul 09. View Abstract
32. Post-acute and Chronic Kidney Function Outcomes of COVID-19 in Children and Adolescents: An EHR Cohort Study from the RECOVER Initiative. medRxiv. 2024 Jun 26. View Abstract
33. Post-Acute Cardiovascular Outcomes of COVID-19 in Children and Adolescents: An EHR Cohort Study from the RECOVER Project. medRxiv. 2024 May 15. View Abstract
34. 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
35. Vaccine Effectiveness Against Long COVID in Children. Pediatrics. 2024 Apr 01; 153(4). View Abstract
36. [Evidence-based interventions to treat chronic low back pain: treatment selection for a personalized medicine approach : German version]. Schmerz. 2025 Dec; 39(6):436-446. View Abstract
37. Validation of a simple body map to measure widespread pain in urologic chronic pelvic pain syndrome: A MAPP Research Network study. Neurourol Urodyn. 2024 Mar; 43(3):727-737. View Abstract
38. Limitations of Noninvasive Tests-Based Population-Level Risk Stratification Strategy for Nonalcoholic Fatty Liver Disease. Dig Dis Sci. 2024 Feb; 69(2):370-383. View Abstract
39. Clonal selection of hematopoietic stem cells after gene therapy for sickle cell disease. Nat Med. 2023 12; 29(12):3175-3183. View Abstract
40. Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome. Blood. 2023 10 12; 142(15):1281-1296. View Abstract
41. Genetic reversal of the globin switch concurrently modulates both fetal and sickle hemoglobin and reduces red cell sickling. Nat Commun. 2023 09 20; 14(1):5850. View Abstract
42. Predicting chronic postsurgical pain: current evidence and a novel program to develop predictive biomarker signatures. Pain. 2023 09 01; 164(9):1912-1926. View Abstract
43. Gene editing without ex vivo culture evades genotoxicity in human hematopoietic stem cells. bioRxiv. 2023 May 27. View Abstract
44. Expanded phenotypic and hematologic abnormalities beyond bone marrow failure in MECOM-associated syndromes. Am J Med Genet A. 2023 07; 191(7):1826-1835. View Abstract
45. 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
46. 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 Abstract
47. Secondary Neoplasms After Hematopoietic Cell Transplant for Sickle Cell Disease. J Clin Oncol. 2023 04 20; 41(12):2227-2237. View Abstract
48. 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
49. 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
50. Evidence-based interventions to treat chronic low back pain: treatment selection for a personalized medicine approach. Pain Rep. 2022 Sep-Oct; 7(5):e1019. View Abstract
51. The long road traveled in hematopoietic stem cell gene therapy. Mol Ther. 2022 10 05; 30(10):3097-3099. View Abstract
52. Patient-Centered Pain Care Using Artificial Intelligence and Mobile Health Tools: A Randomized Comparative Effectiveness Trial. JAMA Intern Med. 2022 09 01; 182(9):975-983. View Abstract
53. If you personalize it, will they use it?: Self-reported and observed use of a tailored, internet-based pain self-management program. Transl Behav Med. 2022 05 26; 12(5):693-701. View Abstract
54. 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
55. Multi-Site Observational Study to Assess Biomarkers for Susceptibility or Resilience to Chronic Pain: The Acute to Chronic Pain Signatures (A2CPS) Study Protocol. Front Med (Lausanne). 2022; 9:849214. View Abstract
56. 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
57. Synergies between centralized and federated approaches to data quality: a report from the national COVID cohort collaborative. J Am Med Inform Assoc. 2022 03 15; 29(4):609-618. View Abstract
58. Advocacy for research starting early in the life course. Pediatr Res. 2022 05; 91(6):1312-1314. View Abstract
59. Clonal hematopoiesis in sickle cell disease. J Clin Invest. 2022 02 15; 132(4). View Abstract
60. 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
61. Investigational curative gene therapy approaches to sickle cell disease. Blood Adv. 2021 12 14; 5(23):5452. View Abstract
62. Congenital X-linked neutropenia with myelodysplasia and somatic tetraploidy due to a germline mutation in SEPT6. Am J Hematol. 2022 01 01; 97(1):18-29. View Abstract
63. 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 Abstract
64. 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 Abstract
65. 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
66. Predictors of engagement in an internet-based cognitive behavioral therapy program for veterans with chronic low back pain. Transl Behav Med. 2021 06 17; 11(6):1274-1282. View Abstract
67. Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease. N Engl J Med. 2021 01 21; 384(3):205-215. View Abstract
68. 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
69. The Changing Face of Adrenoleukodystrophy. Endocr Rev. 2020 08 01; 41(4). View Abstract
70. 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
71. Children's rare disease cohorts: an integrative research and clinical genomics initiative. NPJ Genom Med. 2020; 5:29. View Abstract
72. Children's rare disease cohorts: an integrative research and clinical genomics initiative. NPJ Genom Med. 2020 Jul 06; 5(1):29. View Abstract
73. The Multidisciplinary Approach to The Study of Chronic Pelvic Pain (MAPP) Research Network*: Design and implementation of the Symptom Patterns Study (SPS). Neurourol Urodyn. 2020 08; 39(6):1803-1814. View Abstract
74. HACE1 Prevents Lung Carcinogenesis via Inhibition of RAC-Family GTPases. Cancer Res. 2020 07 15; 80(14):3009-3022. View Abstract
75. Current and future gene therapies for hemoglobinopathies. Curr Opin Hematol. 2020 05; 27(3):149-154. View Abstract
76. Infantile Myelofibrosis and Myeloproliferation with CDC42 Dysfunction. J Clin Immunol. 2020 05; 40(4):554-566. View Abstract
77. 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
78. Rac1/2 activation promotes FGFR1 driven leukemogenesis in stem cell leukemia/lymphoma syndrome. Haematologica. 2020; 105(2):e68-e71. View Abstract
79. Internet-Based Pain Self-Management for Veterans: Feasibility and Preliminary Efficacy of the Pain EASE Program. Pain Pract. 2020 04; 20(4):357-370. View Abstract
80. Lentiviral gene therapy for X-linked chronic granulomatous disease. Nat Med. 2020 02; 26(2):200-206. View Abstract
81. Toxicity and response after CD19-specific CAR T-cell therapy in pediatric/young adult relapsed/refractory B-ALL. Blood. 2019 12 26; 134(26):2361-2368. View Abstract
82. Risankizumab compared with adalimumab in patients with moderate-to-severe plaque psoriasis (IMMvent): a randomised, double-blind, active-comparator-controlled phase 3 trial. Lancet. 2019 08 17; 394(10198):576-586. View Abstract
83. Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study. Haematologica. 2019 10; 104(10):1974-1983. View Abstract
84. Highly efficient therapeutic gene editing of human hematopoietic stem cells. Nat Med. 2019 May; 25(5):776-783. View Abstract
85. Perioperative Quality Initiative consensus statement on preoperative blood pressure, risk and outcomes for elective surgery. Br J Anaesth. 2019 May; 122(5):552-562. View Abstract
86. 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
87. 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
88. 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 Abstract
89. Adverse Childhood Experiences and Symptoms of Urologic Chronic Pelvic Pain Syndrome: A Multidisciplinary Approach to the Study of Chronic Pelvic Pain Research Network Study. Ann Behav Med. 2018 09 13; 52(10):865-877. View Abstract
90. The pseudokinase MLKL activates PAD4-dependent NET formation in necroptotic neutrophils. Sci Signal. 2018 09 04; 11(546). View Abstract
91. 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
92. Long-term adalimumab efficacy in patients with moderate-to-severe hidradenitis suppurativa/acne inversa: 3-year results of a phase 3 open-label extension study. J Am Acad Dermatol. 2019 Jan; 80(1):60-69.e2. View Abstract
93. Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2018 02 01; 378(5):490-491. View Abstract
94. 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
95. 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 Abstract
96. 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
97. Adalimumab for nail psoriasis: Efficacy and safety from the first 26 weeks of a phase 3, randomized, placebo-controlled trial. J Am Acad Dermatol. 2018 Jan; 78(1):90-99.e1. View Abstract
98. Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2017 10 26; 377(17):1630-1638. View Abstract
99. 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 Abstract
100. The 2017 ASPHO distinguished career award goes to Holcombe E. Grier, MD. Pediatr Blood Cancer. 2017 06; 64 Suppl 1. View Abstract
101. Evolving Gene Therapy in Primary Immunodeficiency. Mol Ther. 2017 05 03; 25(5):1132-1141. View Abstract
102. Inhibition of the Proteasome ß2 Site Sensitizes Triple-Negative Breast Cancer Cells to ß5 Inhibitors and Suppresses Nrf1 Activation. Cell Chem Biol. 2017 Feb 16; 24(2):218-230. View Abstract
103. Rapid Lentiviral Transduction Preserves the Engraftment Potential of Fanca-/- Hematopoietic Stem Cells. Mol Ther. 2008 Jun; 16(6):1154-1160. View Abstract
104. AAV and Insertional Mutagenesis. Mol Ther. 2007 Oct; 15(10):1737. View Abstract
105. Of Mouse Models and Men. Mol Ther. 2007 Apr; 15(4):643. View Abstract
106. Molecular Therapy Moves on to a New Editor-in-Chief. Mol Ther. 2009 Dec; 17(12):1989-1990. View Abstract
107. Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype. J Clin Invest. 2016 10 03; 126(10):3868-3878. View Abstract
108. Assessment of physical function and participation in chronic pain clinical trials: IMMPACT/OMERACT recommendations. Pain. 2016 Sep; 157(9):1836-1850. View Abstract
109. 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
110. Two Phase 3 Trials of Adalimumab for Hidradenitis Suppurativa. N Engl J Med. 2016 Aug 04; 375(5):422-34. View Abstract
111. Mathematical modeling of erythrocyte chimerism informs genetic intervention strategies for sickle cell disease. Am J Hematol. 2016 09; 91(9):931-7. View Abstract
112. 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
113. Alpharetroviral self-inactivating vectors produced by a superinfection-resistant stable packaging cell line allow genetic modification of primary human T lymphocytes. Biomaterials. 2016 08; 97:97-109. View Abstract
114. Mechanism of Drug-Drug Interactions Between Warfarin and Statins. J Pharm Sci. 2016 06; 105(6):1976-1986. View Abstract
115. 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
116. 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
117. 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
118. 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
119. Flow cytometric assay for direct quantification of neutrophil extracellular traps in blood samples. Am J Hematol. 2015 Dec; 90(12):1155-8. View Abstract
120. miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction. Mol Ther. 2015 Sep; 23(9):1465-74. View Abstract
121. Perivascular deletion of murine Rac reverses the ratio of marrow arterioles and sinusoid vessels and alters hematopoiesis in vivo. Blood. 2015 May 14; 125(20):3105-13. View Abstract
122. Exit from dormancy provokes DNA-damage-induced attrition in haematopoietic stem cells. Nature. 2015 Apr 23; 520(7548):549-52. View Abstract
123. Germline ETV6 mutations in familial thrombocytopenia and hematologic malignancy. Nat Genet. 2015 Feb; 47(2):180-5. View Abstract
124. RAC2 loss-of-function mutation in 2 siblings with characteristics of common variable immunodeficiency. J Allergy Clin Immunol. 2015 May; 135(5):1380-4.e1-5. View Abstract
125. 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
126. A modified ?-retrovirus vector for X-linked severe combined immunodeficiency. N Engl J Med. 2014 Oct 09; 371(15):1407-17. View Abstract
127. Genomic analysis of bone marrow failure and myelodysplastic syndromes reveals phenotypic and diagnostic complexity. Haematologica. 2015 Jan; 100(1):42-8. View Abstract
128. Unexpected help: mTOR meets lentiviral vectors. Blood. 2014 Aug 07; 124(6):832-3. View Abstract
129. Modulating the stem cell niche for tissue regeneration. Nat Biotechnol. 2014 Aug; 32(8):795-803. View Abstract
130. 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
131. Charting a clear path: the ASGCT Standardized Pathways Conference. Mol Ther. 2014 Jul; 22(7):1235-1238. View Abstract
132. Aberrant overexpression of CD14 on granulocytes sensitizes the innate immune response in mDia1 heterozygous del(5q) MDS. Blood. 2014 Jul 31; 124(5):780-90. View Abstract
133. 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
134. 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
135. Retroviral transduction of murine and human hematopoietic progenitors and stem cells. Methods Mol Biol. 2014; 1185:287-309. View Abstract
136. Curing genetic disease with gene therapy. Trans Am Clin Climatol Assoc. 2014; 125:122-8; discussion 128-9. View Abstract
137. 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
138. 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
139. Oxandrolone for the treatment of bone marrow failure in Fanconi anemia. Pediatr Blood Cancer. 2014 Jan; 61(1):11-9. View Abstract
140. Broadening the indications for hematopoietic stem cell genetic therapies. Cell Stem Cell. 2013 Sep 05; 13(3):263-4. View Abstract
141. 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
142. 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
143. Enough is indeed enough: ACGME required changes in pediatric training. Pediatr Blood Cancer. 2012 Dec 15; 59(7):1158-9. View Abstract
144. 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
145. Vav3 collaborates with p190-BCR-ABL in lymphoid progenitor leukemogenesis, proliferation, and survival. Blood. 2012 Jul 26; 120(4):800-11. View Abstract
146. CTC1 Mutations in a patient with dyskeratosis congenita. Pediatr Blood Cancer. 2012 Aug; 59(2):311-4. View Abstract
147. 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
148. Signaling and cytoskeletal requirements in erythroblast enucleation. Blood. 2012 Jun 21; 119(25):6118-27. View Abstract
149. Overcoming reprogramming resistance of Fanconi anemia cells. Blood. 2012 Jun 07; 119(23):5449-57. View Abstract
150. In vivo enrichment of cytidine deaminase gene-modified hematopoietic cells by prolonged cytosine-arabinoside application. Cytotherapy. 2012 Apr; 14(4):451-60. View Abstract
151. 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
152. PTEN negatively regulates engulfment of apoptotic cells by modulating activation of Rac GTPase. J Immunol. 2011 Dec 01; 187(11):5783-94. View Abstract
153. 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
154. Differential niche and Wnt requirements during acute myeloid leukemia progression. Blood. 2011 Sep 08; 118(10):2849-56. View Abstract
155. 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
156. 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
157. Out of harm's way. Nat Biotechnol. 2011 Jan; 29(1):41-2. View Abstract
158. RhoH regulates subcellular localization of ZAP-70 and Lck in T cell receptor signaling. PLoS One. 2010 Nov 12; 5(11):e13970. View Abstract
159. Transatlantic consortium spotlights need for changes in gene therapy trials. Mol Ther. 2010 Nov; 18(11):1892. View Abstract
160. 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
161. 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
162. 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
163. Rac2 GTPase deficiency depletes BCR-ABL+ leukemic stem cells and progenitors in vivo. Blood. 2010 Jul 08; 116(1):81-4. View Abstract
164. 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
165. Rac GTPases in human diseases. Dis Markers. 2010; 29(3-4):177-87. View Abstract
166. Hematology grants workshop. Hematology Am Soc Hematol Educ Program. 2010; 2010:189-90. View Abstract
167. 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
168. Correction and apology. Mol Ther. 2009 Oct; 17(10):1660. View Abstract
169. National Institutes of Health releases new guidelines on human stem cell research. Mol Ther. 2009 Sep; 17(9):1485-6. View Abstract
170. Loss of the Rho GTPase activating protein p190-B enhances hematopoietic stem cell engraftment potential. Blood. 2009 Oct 22; 114(17):3557-66. View Abstract
171. Gene therapy continues to mature and to face challenges. Mol Ther. 2009 Aug; 17(8):1305-6. View Abstract
172. Rho GTPases in hematopoietic stem cell functions. Curr Opin Hematol. 2009 Jul; 16(4):249-54. View Abstract
173. Identification and characterization of mutations in FANCL gene: a second case of Fanconi anemia belonging to FA-L complementation group. Hum Mutat. 2009 Jul; 30(7):E761-70. View Abstract
174. Rapid development of pluripotent stem cells as a potential therapeutic modality. Mol Ther. 2009 Jun; 17(6):929-30. View Abstract
175. Impaired FANCD2 monoubiquitination and hypersensitivity to camptothecin uniquely characterize Fanconi anemia complementation group M. Blood. 2009 Jul 02; 114(1):174-80. View Abstract
176. 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
177. 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
178. Progress in genetic therapy for severe combined immunodeficiency associated with adenosine deaminase deficiency. Mol Ther. 2009 Apr; 17(4):577-8. View Abstract
179. Upping the ante: recent advances in direct reprogramming. Mol Ther. 2009 Jun; 17(6):947-53. View Abstract
180. 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
181. New approaches in the potential treatment of HIV-acquired immunodeficiency disease. Mol Ther. 2009 Feb; 17(2):209-10. View Abstract
182. ESCGT 2008: progress in clinical gene therapy. Mol Ther. 2009 Jan; 17(1):1-2. View Abstract
183. 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
184. Sleeping beauty vector system moves toward human trials in the United States. Mol Ther. 2008 Sep; 16(9):1515-6. View Abstract
185. 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 Abstract
186. Progress reported in two studies of clinical gene transfer into the retina. Mol Ther. 2008 Jul; 16(7):1181. View Abstract
187. 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
188. Defective homing is associated with altered Cdc42 activity in cells from patients with Fanconi anemia group A. Blood. 2008 Sep 01; 112(5):1683-6. View Abstract
189. A "vector drain" in US gene therapy development? Mol Ther. 2008 May; 16(5):801-2. View Abstract
190. Rapid lentiviral transduction preserves the engraftment potential of Fanca(-/-) hematopoietic stem cells. Mol Ther. 2008 Jun; 16(6):1154-60. View Abstract
191. Foamy virus vectors come of age. Mol Ther. 2008 Apr; 16(4):635-6. View Abstract
192. 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
193. Rho GTPases and regulation of hematopoietic stem cell localization. Methods Enzymol. 2008; 439:365-93. View Abstract
194. Cross-talk between RhoH and Rac1 in regulation of actin cytoskeleton and chemotaxis of hematopoietic progenitor cells. Blood. 2008 Mar 01; 111(5):2597-605. View Abstract
195. 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
196. 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
197. RAC reviews serious adverse event associated with AAV therapy trial. Mol Ther. 2007 Dec; 15(12):2053-4. View Abstract
198. Rac guanosine triphosphatases represent integrating molecular therapeutic targets for BCR-ABL-induced myeloproliferative disease. Cancer Cell. 2007 Nov; 12(5):467-78. View Abstract
199. TNF-alpha induces leukemic clonal evolution ex vivo in Fanconi anemia group C murine stem cells. J Clin Invest. 2007 Nov; 117(11):3283-95. View Abstract
200. Cdc42 critically regulates the balance between myelopoiesis and erythropoiesis. Blood. 2007 Dec 01; 110(12):3853-61. View Abstract
201. Small pituitary size in children with Fanconi anemia. Pediatr Blood Cancer. 2007 Aug; 49(2):166-70. View Abstract
202. A new mechanism of leukemia drug resistance? N Engl J Med. 2007 Jul 05; 357(1):77-8. View Abstract
203. NIH decides against continuing NGVLs in their current form. Mol Ther. 2007 Jul; 15(7):1223. View Abstract
204. Chemotherapy for myeloid malignancy in children with Fanconi anemia. Pediatr Blood Cancer. 2007 Jun 15; 48(7):668-72. View Abstract
205. 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
206. Inflammatory reactive oxygen species-mediated hemopoietic suppression in Fancc-deficient mice. J Immunol. 2007 Apr 15; 178(8):5277-87. View Abstract
207. Inflammatory ROS promote and cooperate with the Fanconi anemia mutation for hematopoietic senescence. J Cell Sci. 2007 May 01; 120(Pt 9):1572-83. View Abstract
208. Are lentivirus vectors safer? Mol Ther. 2007 Mar; 15(3):439. View Abstract
209. 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
210. ASGT advises NIH on funding of gene therapy trials. Mol Ther. 2007 Jan; 15(1):1-2. View Abstract
211. Stem cell collection and gene transfer in Fanconi anemia. Mol Ther. 2007 Jan; 15(1):211-9. View Abstract
212. Rac1 GTPase regulates cell genomic stability and senescence. J Biol Chem. 2006 Dec 15; 281(50):38519-28. View Abstract
213. RhoH GTPase recruits and activates Zap70 required for T cell receptor signaling and thymocyte development. Nat Immunol. 2006 Nov; 7(11):1182-90. View Abstract
214. Children are not little adults: just ask their hematopoietic stem cells. J Clin Invest. 2006 Oct; 116(10):2593-6. View Abstract
215. NIH funding of gene therapy trials. Mol Ther. 2006 Nov; 14(5):607. View Abstract
216. Vector insertion, mutagenesis and transgene toxicity. Mol Ther. 2006 Oct; 14(4):457. View Abstract
217. Rac GTPases regulate the morphology and deformability of the erythrocyte cytoskeleton. Blood. 2006 Dec 01; 108(12):3637-45. View Abstract
218. The role of chemokine activation of Rac GTPases in hematopoietic stem cell marrow homing, retention, and peripheral mobilization. Exp Hematol. 2006 Aug; 34(8):976-85. View Abstract
219. A pox on your tumor. Mol Ther. 2006 Sep; 14(3):313. View Abstract
220. Inhibition of RhoA GTPase activity enhances hematopoietic stem and progenitor cell proliferation and engraftment. Blood. 2006 Sep 15; 108(6):2087-94. View Abstract
221. Genetic deletion of Rac1 GTPase reveals its critical role in actin stress fiber formation and focal adhesion complex assembly. J Biol Chem. 2006 Jul 07; 281(27):18652-9. View Abstract
222. Gene therapy advances but struggles to interpret safety data in small animal models. Mol Ther. 2006 Jun; 13(6):1027-8. View Abstract
223. Lentiviral vectors pseudotyped with murine ecotropic envelope: increased biosafety and convenience in preclinical research. Exp Hematol. 2006 May; 34(5):588-92. View Abstract
224. Adventitious mutations in clinical grade vectors: an issue to consider? Mol Ther. 2006 May; 13(5):831-2. View Abstract
225. An emerging consensus on recommendations to facilitate clinical gene transfer. Mol Ther. 2006 Apr; 13(4):637-8. View Abstract
226. HOXB4 inhibits cell growth in a dose-dependent manner and sensitizes cells towards extrinsic cues. Cell Cycle. 2006 Jan; 5(1):14-22. View Abstract
227. Structure-function based design of small molecule inhibitors targeting Rho family GTPases. Curr Top Med Chem. 2006; 6(11):1109-16. View Abstract
228. Rational design and applications of a Rac GTPase-specific small molecule inhibitor. Methods Enzymol. 2006; 406:554-65. View Abstract
229. 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
230. 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
231. New AAV serotypes may broaden the therapeutic pipeline to human gene therapy. Mol Ther. 2006 Jan; 13(1):1-2. View Abstract
232. FDA guidance document on monitoring delayed adverse events a good first start. Mol Ther. 2005 Dec; 12(6):991-2. View Abstract
233. Promoting translational research in academic health centers: navigating the "roadmap". Acad Med. 2005 Nov; 80(11):1012-8. View Abstract
234. 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
235. 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
236. Genetic deletion of Cdc42GAP reveals a role of Cdc42 in erythropoiesis and hematopoietic stem/progenitor cell survival, adhesion, and engraftment. Blood. 2006 Jan 01; 107(1):98-105. View Abstract
237. A rapid method for retrovirus-mediated identification of complementation groups in Fanconi anemia patients. Mol Ther. 2005 Nov; 12(5):976-84. View Abstract
238. Rac GTPases differentially integrate signals regulating hematopoietic stem cell localization. Nat Med. 2005 Aug; 11(8):886-91. View Abstract
239. A balanced decision? Regulatory reaction to the "third case". Mol Ther. 2005 Jun; 11(6):819-20. View Abstract
240. Rac2 regulates neutrophil chemotaxis, superoxide production, and myeloid colony formation through multiple distinct effector pathways. J Immunol. 2005 Apr 15; 174(8):4613-20. View Abstract
241. Rac2-deficient hematopoietic stem cells show defective interaction with the hematopoietic microenvironment and long-term engraftment failure. Stem Cells. 2005 Mar; 23(3):335-46. View Abstract
242. RhoH GTPase: a key regulator of hematopoietic cell proliferation and apoptosis? Cell Cycle. 2005 Feb; 4(2):201-2. View Abstract
243. The NIH roadmap: timing is everything. Mol Ther. 2005 Feb; 11(2):173. View Abstract
244. 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
245. Lenti in red: progress in gene therapy for human hemoglobinopathies. J Clin Invest. 2004 Oct; 114(7):889-91. View Abstract
246. Localization of Rac2 via the C terminus and aspartic acid 150 specifies superoxide generation, actin polarity and chemotaxis in neutrophils. Nat Immunol. 2004 Jul; 5(7):744-51. View Abstract
247. Chance or necessity? Insertional mutagenesis in gene therapy and its consequences. Mol Ther. 2004 Jan; 9(1):5-13. View Abstract
248. 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
249. Medicine. Gene therapy--new challenges ahead. Science. 2003 Oct 17; 302(5644):400-1. View Abstract
250. Hematopoietic cell regulation by Rac1 and Rac2 guanosine triphosphatases. Science. 2003 Oct 17; 302(5644):445-9. View Abstract
251. 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
252. 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 Abstract
253. Side effects of retroviral gene transfer into hematopoietic stem cells. Blood. 2003 Mar 15; 101(6):2099-114. View Abstract
254. Rac2, a hematopoiesis-specific Rho GTPase, specifically regulates mast cell protease gene expression in bone marrow-derived mast cells. Mol Cell Biol. 2002 Nov; 22(21):7645-57. View Abstract
255. 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 Abstract
256. Gene Therapy 2000. Hematology Am Soc Hematol Educ Program. 2000; 376-393. View Abstract
257. Mouse model of X-linked chronic granulomatous disease, an inherited defect in phagocyte superoxide production. Nat Genet. 1995 Feb; 9(2):202-9. View Abstract
258. Novel human oncogene lbc detected by transfection with distinct homology regions to signal transduction products. Oncogene. 1994 Feb; 9(2):621-8. View Abstract
259. Long-term in vivo expression of a murine adenosine deaminase gene in rhesus monkey hematopoietic cells of multiple lineages after retroviral mediated gene transfer into CD34+ bone marrow cells. Blood. 1993 Oct 01; 82(7):1975-80. View Abstract
260. Restriction of neuroblastoma to the prostate gland in transgenic mice. Mol Cell Biol. 1991 Sep; 11(9):4518-27. View Abstract
261. Retroviral gene transfer of human adenosine deaminase in murine hematopoietic cells: effect of selectable marker sequences on long-term expression. Blood. 1991 Jul 15; 78(2):310-7. View Abstract
262. Gene transfer into murine hematopoietic stem cells and bone marrow stromal cells. Ann N Y Acad Sci. 1990; 612:398-406. View Abstract
263. Long-term expression of human adenosine deaminase in mice transplanted with retrovirus-infected hematopoietic stem cells. Proc Natl Acad Sci U S A. 1989 Nov; 86(22):8892-6. View Abstract
264. Gene therapy of somatic cells: status and prospects. Prog Med Genet. 1988; 7:130-42. View Abstract
265. Retrovirus-mediated gene transfer of human adenosine deaminase: expression of functional enzyme in murine hematopoietic stem cells in vivo. Mol Cell Biol. 1987 Oct; 7(10):3459-65. View Abstract
266. Transfer and expression of human ADA in murine hematopoietic stem cells. Prog Clin Biol Res. 1987; 251:567-80. View Abstract
267. Retrovirus-mediated transfer of human adenosine deaminase gene sequences into cells in culture and into murine hematopoietic cells in vivo. Proc Natl Acad Sci U S A. 1986 Apr; 83(8):2566-70. View Abstract
268. Somatic gene therapy. Current status and future prospects. J Clin Invest. 1986 Apr; 77(4):1053-6. View Abstract
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