Harvard Catalyst Profiles

Contact, publication, and social network information about Harvard faculty and fellows.

David Allan Williams, M.D.


The research activities and funding listed below are automatically derived from NIH ExPORTER and other sources, which might result in incorrect or missing items. Faculty can login to make corrections and additions.
  1. OT2HL152639 (WILLIAMS, DAVID A) Sep 20, 2019 - Feb 17, 2020
    Boston Consortium to Cure SCD - Williams/John Higgins
    Role: Principal Investigator
  2. OT2HL152640 (WILLIAMS, DAVID A) Sep 20, 2019 - Feb 17, 2020
    Boston Consortium to Cure SCD - Williams/Ellen Grant
    Role: Principal Investigator
  3. R01HL137848 (WILLIAMS, DAVID A) Sep 5, 2017 - May 31, 2022
    Gene therapy targeting BCL11A to induce fetal hemoglobin and reduce sickle hemoglobin in patients with Sickle Cell Disease
    Role: Principal Investigator
  4. R56AI125766 (SNAPPER, SCOTT B) Aug 8, 2017 - Jul 31, 2018
    Employing novel humanized murine strains to develop hematopoietic stem cell gene therapeutic approaches for very early-onset inflammatory bowel disease due to IL10-receptor deficiency.
    Role: Co-Principal Investigator
  5. R34HL133384 (WILLIAMS, DAVID A) Apr 10, 2017 - Mar 31, 2020
    Feasibility study randomizing IST vs URD BMT for children with aplastic anemia
    Role: Principal Investigator

Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Faculty can login to make corrections and additions.
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PMC Citations indicate the number of times the publication was cited by articles in PubMed Central, and the Altmetric score represents citations in news articles and social media. (Note that publications are often cited in additional ways that are not shown here.) Fields are based on how the National Library of Medicine (NLM) classifies the publication's journal and might not represent the specific topic of the publication. Translation tags are based on the publication type and the MeSH terms NLM assigns to the publication. Some publications (especially newer ones and publications not in PubMed) might not yet be assigned Field or Translation tags.) Click a Field or Translation tag to filter the publications.
  1. Wu Y, Zeng J, Roscoe BP, Liu P, Yao Q, Lazzarotto CR, Clement K, Cole MA, Luk K, Baricordi C, Shen AH, Ren C, Esrick EB, Manis JP, Dorfman DM, Williams DA, Biffi A, Brugnara C, Biasco L, Brendel C, Pinello L, Tsai SQ, Wolfe SA, Bauer DE. Highly efficient therapeutic gene editing of human hematopoietic stem cells. Nat Med. 2019 05; 25(5):776-783. PMID: 30911135.
  2. Williams DA. 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. PMID: 30773447.
  3. Esrick EB, Manis JP, Daley H, Baricordi C, Trébéden-Negre H, Pierciey FJ, Armant M, Nikiforow S, Heeney MM, London WB, Biasco L, Asmal M, Williams DA, Biffi A. Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients. Blood Adv. 2018 10 09; 2(19):2505-2512. PMID: 30282642.
  4. D'Cruz AA, Speir M, Bliss-Moreau M, Dietrich S, Wang S, Chen AA, Gavillet M, Al-Obeidi A, Lawlor KE, Vince JE, Kelliher MA, Hakem R, Pasparakis M, Williams DA, Ericsson M, Croker BA. The pseudokinase MLKL activates PAD4-dependent NET formation in necroptotic neutrophils. Sci Signal. 2018 09 04; 11(546). PMID: 30181240.
    Citations:    Fields:    
  5. Park HH, Triboulet R, Bentler M, Guda S, Du P, Xu H, Gregory RI, Brendel C, Williams DA. DROSHA Knockout Leads to Enhancement of Viral Titers for Vectors Encoding miRNA-Adapted shRNAs. Mol Ther Nucleic Acids. 2018 Sep 07; 12:591-599. PMID: 30195795.
  6. Williams DA, Eichler F, Duncan C. Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2018 02 01; 378(5):490-491. PMID: 29385368.
    Citations:    Fields:    Translation:Humans
  7. De Vita S, Li Y, Harris CE, McGuinness MK, Ma C, Williams DA. 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. PMID: 29235178.
    Citations:    Fields:    Translation:AnimalsCells
  8. Gavillet M, Martinod K, Renella R, Wagner DD, Williams DA. 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. PMID: 29124783.
    Citations: 2     Fields:    Translation:AnimalsCells
  9. Eichler F, Duncan C, Musolino PL, Orchard PJ, De Oliveira S, Thrasher AJ, Armant M, Dansereau C, Lund TC, Miller WP, Raymond GV, Sankar R, Shah AJ, Sevin C, Gaspar HB, Gissen P, Amartino H, Bratkovic D, Smith NJC, Paker AM, Shamir E, O'Meara T, Davidson D, Aubourg P, Williams DA. Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2017 10 26; 377(17):1630-1638. PMID: 28976817.
    Citations: 11     Fields:    Translation:HumansCellsCTClinical Trials
  10. Williams DA, Orkin SH. The 2017 ASPHO distinguished career award goes to Holcombe E. Grier, MD. Pediatr Blood Cancer. 2017 06; 64 Suppl 1. PMID: 28371236.
    Citations:    Fields:    
  11. Thrasher AJ, Williams DA. Evolving Gene Therapy in Primary Immunodeficiency. Mol Ther. 2017 05 03; 25(5):1132-1141. PMID: 28366768.
  12. Müller LU, Milsom MD, Kim MO, Schambach A, Schuesler T, Williams DA. Rapid Lentiviral Transduction Preserves the Engraftment Potential of Fanca-/- Hematopoietic Stem Cells. Mol Ther. 2008 Jun; 16(6):1154-1160. PMID: 28178472.
    Citations:    Fields:    
  13. Williams DA. Of Mouse Models and Men. Mol Ther. 2007 Apr; 15(4):643. PMID: 28192701.
  14. Williams DA. AAV and Insertional Mutagenesis. Mol Ther. 2007 Oct; 15(10):1737. PMID: 28182914.
  15. Williams DA. Molecular Therapy Moves on to a New Editor-in-Chief. Mol Ther. 2009 Dec; 17(12):1989-1990. PMID: 28178450.
  16. Brendel C, Guda S, Renella R, Bauer DE, Canver MC, Kim YJ, Heeney MM, Klatt D, Fogel J, Milsom MD, Orkin SH, Gregory RI, Williams DA. Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype. J Clin Invest. 2016 10 03; 126(10):3868-3878. PMID: 27599293.
    Citations: 10     Fields:    Translation:HumansAnimalsCells
  17. Taylor AM, Phillips K, Patel KV, Turk DC, Dworkin RH, Beaton D, Clauw DJ, Gignac MA, Markman JD, Williams DA, Bujanover S, Burke LB, Carr DB, Choy EH, Conaghan PG, Cowan P, Farrar JT, Freeman R, Gewandter J, Gilron I, Goli V, Gover TD, Haddox JD, Kerns RD, Kopecky EA, Lee DA, Malamut R, Mease P, Rappaport BA, Simon LS, Singh JA, Smith SM, Strand V, Tugwell P, Vanhove GF, Veasley C, Walco GA, Wasan AD, Witter J. Assessment of physical function and participation in chronic pain clinical trials: IMMPACT/OMERACT recommendations. Pain. 2016 Sep; 157(9):1836-50. PMID: 27058676.
    Citations: 11     Fields:    Translation:Humans
  18. Mino A, Troeger A, Brendel C, Cantor A, Harris C, Ciuculescu MF, Williams DA. 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. PMID: 27574848.
    Citations:    Fields:    Translation:HumansCells
  19. Kimball AB, Okun MM, Williams DA, Gottlieb AB, Papp KA, Zouboulis CC, Armstrong AW, Kerdel F, Gold MH, Forman SB, Korman NJ, Giamarellos-Bourboulis EJ, Crowley JJ, Lynde C, Reguiai Z, Prens EP, Alwawi E, Mostafa NM, Pinsky B, Sundaram M, Gu Y, Carlson DM, Jemec GB. Two Phase 3 Trials of Adalimumab for Hidradenitis Suppurativa. N Engl J Med. 2016 Aug 04; 375(5):422-34. PMID: 27518661.
    Citations: 23     Fields:    Translation:HumansCTClinical Trials
  20. Altrock PM, Brendel C, Renella R, Orkin SH, Williams DA, Michor F. Mathematical modeling of erythrocyte chimerism informs genetic intervention strategies for sickle cell disease. Am J Hematol. 2016 09; 91(9):931-7. PMID: 27299299.
    Citations: 8     Fields:    Translation:HumansAnimalsCells
  21. Townsend EC, Murakami MA, Christodoulou A, Christie AL, Köster J, DeSouza TA, Morgan EA, Kallgren SP, Liu H, Wu SC, Plana O, Montero J, Stevenson KE, Rao P, Vadhi R, Andreeff M, Armand P, Ballen KK, Barzaghi-Rinaudo P, Cahill S, Clark RA, Cooke VG, Davids MS, DeAngelo DJ, Dorfman DM, Eaton H, Ebert BL, Etchin J, Firestone B, Fisher DC, Freedman AS, Galinsky IA, Gao H, Garcia JS, Garnache-Ottou F, Graubert TA, Gutierrez A, Halilovic E, Harris MH, Herbert ZT, Horwitz SM, Inghirami G, Intlekofer AM, Ito M, Izraeli S, Jacobsen ED, Jacobson CA, Jeay S, Jeremias I, Kelliher MA, Koch R, Konopleva M, Kopp N, Kornblau SM, Kung AL, Kupper TS, LeBoeuf NR, LaCasce AS, Lees E, Li LS, Look AT, Murakami M, Muschen M, Neuberg D, Ng SY, Odejide OO, Orkin SH, Paquette RR, Place AE, Roderick JE, Ryan JA, Sallan SE, Shoji B, Silverman LB, Soiffer RJ, Steensma DP, Stegmaier K, Stone RM, Tamburini J, Thorner AR, van Hummelen P, Wadleigh M, Wiesmann M, Weng AP, Wuerthner JU, Williams DA, Wollison BM, Lane AA, Letai A, Bertagnolli MM, Ritz J, Brown M, Long H, Aster JC, Shipp MA, Griffin JD, Weinstock DM. The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice. Cancer Cell. 2016 07 11; 30(1):183. PMID: 27479034.
    Citations: 13     Fields:    
  22. Shaik AN, Bohnert T, Williams DA, Gan LL, LeDuc BW. Mechanism of Drug-Drug Interactions Between Warfarin and Statins. J Pharm Sci. 2016 06; 105(6):1976-1986. PMID: 27103011.
    Citations: 7     Fields:    Translation:HumansCells
  23. Townsend EC, Murakami MA, Christodoulou A, Christie AL, Köster J, DeSouza TA, Morgan EA, Kallgren SP, Liu H, Wu SC, Plana O, Montero J, Stevenson KE, Rao P, Vadhi R, Andreeff M, Armand P, Ballen KK, Barzaghi-Rinaudo P, Cahill S, Clark RA, Cooke VG, Davids MS, DeAngelo DJ, Dorfman DM, Eaton H, Ebert BL, Etchin J, Firestone B, Fisher DC, Freedman AS, Galinsky IA, Gao H, Garcia JS, Garnache-Ottou F, Graubert TA, Gutierrez A, Halilovic E, Harris MH, Herbert ZT, Horwitz SM, Inghirami G, Intlekofer AM, Ito M, Izraeli S, Jacobsen ED, Jacobson CA, Jeay S, Jeremias I, Kelliher MA, Koch R, Konopleva M, Kopp N, Kornblau SM, Kung AL, Kupper TS, LeBoeuf NR, LaCasce AS, Lees E, Li LS, Look AT, Murakami M, Muschen M, Neuberg D, Ng SY, Odejide OO, Orkin SH, Paquette RR, Place AE, Roderick JE, Ryan JA, Sallan SE, Shoji B, Silverman LB, Soiffer RJ, Steensma DP, Stegmaier K, Stone RM, Tamburini J, Thorner AR, van Hummelen P, Wadleigh M, Wiesmann M, Weng AP, Wuerthner JU, Williams DA, Wollison BM, Lane AA, Letai A, Bertagnolli MM, Ritz J, Brown M, Long H, Aster JC, Shipp MA, Griffin JD, Weinstock DM. The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice. Cancer Cell. 2016 04 11; 29(4):574-586. PMID: 27070704.
    Citations: 19     Fields:    Translation:HumansAnimalsCells
  24. Reddy PN, Radu M, Xu K, Wood J, Harris CE, Chernoff J, Williams DA. 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. PMID: 26932803.
    Citations: 3     Fields:    Translation:AnimalsCells
  25. Williams DA, Gavillet M. 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. PMID: 26749259.
    Citations:    Fields:    Translation:HumansAnimalsCells
  26. Shaik AN, Grater R, Lulla M, Williams DA, Gan LL, Bohnert T, LeDuc BW. 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. PMID: 26655108.
    Citations: 4     Fields:    Translation:Humans
  27. Gavillet M, Martinod K, Renella R, Harris C, Shapiro NI, Wagner DD, Williams DA. Flow cytometric assay for direct quantification of neutrophil extracellular traps in blood samples. Am J Hematol. 2015 Dec; 90(12):1155-8. PMID: 26347989.
    Citations: 9     Fields:    Translation:HumansAnimalsCells
  28. Guda S, Brendel C, Renella R, Du P, Bauer DE, Canver MC, Grenier JK, Grimson AW, Kamran SC, Thornton J, de Boer H, Root DE, Milsom MD, Orkin SH, Gregory RI, Williams DA. miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction. Mol Ther. 2015 Sep; 23(9):1465-74. PMID: 26080908.
    Citations: 25     Fields:    Translation:HumansAnimalsCells
  29. Bruedigam C, Bagger FO, Heidel FH, Paine Kuhn C, Guignes S, Song A, Austin R, Vu T, Lee E, Riyat S, Moore AS, Lock RB, Bullinger L, Hill GR, Armstrong SA, Williams DA, Lane SW. 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. PMID: 25479751.
    Citations: 18     Fields:    Translation:HumansAnimalsCells
  30. Hacein-Bey-Abina S, Pai SY, Gaspar HB, Armant M, Berry CC, Blanche S, Bleesing J, Blondeau J, de Boer H, Buckland KF, Caccavelli L, Cros G, De Oliveira S, Fernández KS, Guo D, Harris CE, Hopkins G, Lehmann LE, Lim A, London WB, van der Loo JC, Malani N, Male F, Malik P, Marinovic MA, McNicol AM, Moshous D, Neven B, Oleastro M, Picard C, Ritz J, Rivat C, Schambach A, Shaw KL, Sherman EA, Silberstein LE, Six E, Touzot F, Tsytsykova A, Xu-Bayford J, Baum C, Bushman FD, Fischer A, Kohn DB, Filipovich AH, Notarangelo LD, Cavazzana M, Williams DA, Thrasher AJ. A modified ?-retrovirus vector for X-linked severe combined immunodeficiency. N Engl J Med. 2014 Oct 09; 371(15):1407-17. PMID: 25295500.
    Citations: 65     Fields:    Translation:HumansAnimalsCellsCTClinical Trials
  31. Brendel C, Williams DA. Unexpected help: mTOR meets lentiviral vectors. Blood. 2014 Aug 07; 124(6):832-3. PMID: 25104861.
    Citations: 1     Fields:    Translation:HumansAnimalsCells
  32. Renella R, Schlehe JS, Selkoe DJ, Williams DA, LaVoie MJ. 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. PMID: 25043722.
    Citations: 4     Fields:    Translation:AnimalsCells
  33. Kiem HP, Baum C, Bushman FD, Byrne BJ, Carter BJ, Cavagnaro J, Malech HL, Mendell JR, Naldini LM, Sorrentino BP, Williams DA, Flotte TR. Charting a clear path: the ASGCT Standardized Pathways Conference. Mol Ther. 2014 Jul; 22(7):1235-1238. PMID: 24981438.
  34. De Vita S, Schneider RK, Garcia M, Wood J, Gavillet M, Ebert BL, Gerbaulet A, Roers A, Levine RL, Mullally A, Williams DA. Loss of function of TET2 cooperates with constitutively active KIT in murine and human models of mastocytosis. PLoS One. 2014; 9(5):e96209. PMID: 24788138.
    Citations: 13     Fields:    Translation:HumansAnimalsCells
  35. Williams DA, Thrasher AJ. Concise review: lessons learned from clinical trials of gene therapy in monogenic immunodeficiency diseases. Stem Cells Transl Med. 2014 May; 3(5):636-42. PMID: 24682287.
    Citations: 16     Fields:    Translation:HumansAnimals
  36. Ciuculescu MF, Brendel C, Harris CE, Williams DA. Retroviral transduction of murine and human hematopoietic progenitors and stem cells. Methods Mol Biol. 2014; 1185:287-309. PMID: 25062637.
    Citations: 5     Fields:    Translation:HumansAnimalsCells
  37. Williams DA, Bennett C, Bertuch A, Bessler M, Coates T, Corey S, Dror Y, Huang J, Lipton J, Olson TS, Reiss UM, Rogers ZR, Sieff C, Vlachos A, Walkovich K, Wang W, Shimamura A. 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. PMID: 24285674.
    Citations: 4     Fields:    Translation:Humans
  38. Troeger A, Chae HD, Senturk M, Wood J, Williams DA. 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. PMID: 24189071.
    Citations: 2     Fields:    Translation:HumansAnimalsCells
  39. Rose SR, Kim MO, Korbee L, Wilson KA, Ris MD, Eyal O, Sherafat-Kazemzadeh R, Bollepalli S, Harris R, Jeng MR, Williams DA, Smith FO. Oxandrolone for the treatment of bone marrow failure in Fanconi anemia. Pediatr Blood Cancer. 2014 Jan; 61(1):11-9. PMID: 24019220.
    Citations: 8     Fields:    Translation:HumansCTClinical Trials
  40. Williams DA. Broadening the indications for hematopoietic stem cell genetic therapies. Cell Stem Cell. 2013 Sep 05; 13(3):263-4. PMID: 24012366.
    Citations: 5     Fields:    Translation:HumansCells
  41. Mullally A, Bruedigam C, Poveromo L, Heidel FH, Purdon A, Vu T, Austin R, Heckl D, Breyfogle LJ, Kuhn CP, Kalaitzidis D, Armstrong SA, Williams DA, Hill GR, Ebert BL, Lane SW. 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. PMID: 23487027.
    Citations: 34     Fields:    Translation:HumansAnimalsCells
  42. Dorrance AM, De Vita S, Radu M, Reddy PN, McGuinness MK, Harris CE, Mathieu R, Lane SW, Kosoff R, Milsom MD, Chernoff J, Williams DA. 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. PMID: 23335370.
    Citations: 18     Fields:    Translation:AnimalsCells
  43. Williams DA, Mack J. Enough is indeed enough: ACGME required changes in pediatric training. Pediatr Blood Cancer. 2012 Dec 15; 59(7):1158-9. PMID: 22996805.
    Citations:    Fields:    Translation:Humans
  44. Müller LU, Schlaeger TM, DeVine AL, Williams DA. Induced pluripotent stem cells as a tool for gaining new insights into Fanconi anemia. Cell Cycle. 2012 Aug 15; 11(16):2985-90. PMID: 22825249.
    Citations: 5     Fields:    Translation:HumansAnimalsCells
  45. Keller RB, Gagne KE, Usmani GN, Asdourian GK, Williams DA, Hofmann I, Agarwal S. CTC1 Mutations in a patient with dyskeratosis congenita. Pediatr Blood Cancer. 2012 Aug; 59(2):311-4. PMID: 22532422.
    Citations: 45     Fields:    Translation:HumansCells
  46. Troeger A, Johnson AJ, Wood J, Blum WG, Andritsos LA, Byrd JC, Williams DA. RhoH is critical for cell-microenvironment interactions in chronic lymphocytic leukemia in mice and humans. Blood. 2012 May 17; 119(20):4708-18. PMID: 22474251.
    Citations: 16     Fields:    Translation:HumansAnimalsCells
  47. Konstantinidis DG, Pushkaran S, Johnson JF, Cancelas JA, Manganaris S, Harris CE, Williams DA, Zheng Y, Kalfa TA. Signaling and cytoskeletal requirements in erythroblast enucleation. Blood. 2012 Jun 21; 119(25):6118-27. PMID: 22461493.
    Citations: 34     Fields:    Translation:AnimalsCells
  48. Müller LU, Milsom MD, Harris CE, Vyas R, Brumme KM, Parmar K, Moreau LA, Schambach A, Park IH, London WB, Strait K, Schlaeger T, Devine AL, Grassman E, D'Andrea A, Daley GQ, Williams DA. Overcoming reprogramming resistance of Fanconi anemia cells. Blood. 2012 Jun 07; 119(23):5449-57. PMID: 22371882.
    Citations: 57     Fields:    Translation:HumansAnimalsCells
  49. Brennig S, Rattmann I, Lachmann N, Schambach A, Williams DA, Moritz T. In vivo enrichment of cytidine deaminase gene-modified hematopoietic cells by prolonged cytosine-arabinoside application. Cytotherapy. 2012 Apr; 14(4):451-60. PMID: 22242831.
    Citations: 4     Fields:    Translation:AnimalsCells
  50. Lane SW, De Vita S, Alexander KA, Karaman R, Milsom MD, Dorrance AM, Purdon A, Louis L, Bouxsein ML, Williams DA. 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. PMID: 22123845.
    Citations: 10     Fields:    Translation:AnimalsCells
  51. Mondal S, Ghosh-Roy S, Loison F, Li Y, Jia Y, Harris C, Williams DA, Luo HR. PTEN negatively regulates engulfment of apoptotic cells by modulating activation of Rac GTPase. J Immunol. 2011 Dec 01; 187(11):5783-94. PMID: 22043008.
    Citations: 7     Fields:    Translation:AnimalsCells
  52. Mizukawa B, Wei J, Shrestha M, Wunderlich M, Chou FS, Griesinger A, Harris CE, Kumar AR, Zheng Y, Williams DA, Mulloy JC. 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. PMID: 21940819.
    Citations: 23     Fields:    Translation:HumansAnimalsCells
  53. Lane SW, Wang YJ, Lo Celso C, Ragu C, Bullinger L, Sykes SM, Ferraro F, Shterental S, Lin CP, Gilliland DG, Scadden DT, Armstrong SA, Williams DA. Differential niche and Wnt requirements during acute myeloid leukemia progression. Blood. 2011 Sep 08; 118(10):2849-56. PMID: 21765021.
    Citations: 40     Fields:    Translation:HumansAnimalsCells
  54. Sanchez-Aguilera A, Lee YJ, Lo Celso C, Ferraro F, Brumme K, Mondal S, Kim C, Dorrance A, Luo HR, Scadden DT, Williams DA. 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. PMID: 21606370.
    Citations: 18     Fields:    Translation:AnimalsCells
  55. Tolar J, Adair JE, Antoniou M, Bartholomae CC, Becker PS, Blazar BR, Bueren J, Carroll T, Cavazzana-Calvo M, Clapp DW, Dalgleish R, Galy A, Gaspar HB, Hanenberg H, Von Kalle C, Kiem HP, Lindeman D, Naldini L, Navarro S, Renella R, Rio P, Sevilla J, Schmidt M, Verhoeyen E, Wagner JE, Williams DA, Thrasher AJ. 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. PMID: 21540837.
    Citations: 8     Fields:    Translation:HumansCells
  56. Williams DA, Thrasher AJ, Baum C. Transatlantic consortium spotlights need for changes in gene therapy trials. Mol Ther. 2010 Nov; 18(11):1892. PMID: 21042294.
  57. Williams DA, Porter ES, Lux SE, Grier HE, Mack JW, Orkin SH. 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. PMID: 20730793.
    Citations: 1     Fields:    Translation:Humans
  58. Milsom MD, Williams DA. 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. PMID: 20677209.
    Citations:    Fields:    Translation:HumansCells
  59. Williams DA. 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. PMID: 20229496.
    Citations:    Fields:    
  60. Lane SW, Sykes SM, Al-Shahrour F, Shterental S, Paktinat M, Lo Celso C, Jesneck JL, Ebert BL, Williams DA, Gilliland DG. 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. PMID: 20197553.
    Citations: 38     Fields:    Translation:HumansAnimalsCells
  61. Pai SY, Kim C, Williams DA. Rac GTPases in human diseases. Dis Markers. 2010; 29(3-4):177-87. PMID: 21178276.
    Citations: 18     Fields:    Translation:HumansAnimalsCells
  62. Williams DA. Hematology grants workshop. Hematology Am Soc Hematol Educ Program. 2010; 2010:189-90. PMID: 21239792.
    Citations:    Fields:    
  63. Kalfa TA, Pushkaran S, Zhang X, Johnson JF, Pan D, Daria D, Geiger H, Cancelas JA, Williams DA, Zheng Y. 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. PMID: 20065081.
    Citations: 28     Fields:    Translation:AnimalsCells
  64. Williams DA. Correction and apology. Mol Ther. 2009 Oct; 17(10):1660. PMID: 19789558.
  65. Williams DA. National Institutes of Health releases new guidelines on human stem cell research. Mol Ther. 2009 Sep; 17(9):1485-6. PMID: 19721416.
  66. Williams DA. Gene therapy continues to mature and to face challenges. Mol Ther. 2009 Aug; 17(8):1305-6. PMID: 19644491.
  67. Williams DA. Rapid development of pluripotent stem cells as a potential therapeutic modality. Mol Ther. 2009 Jun; 17(6):929-30. PMID: 19483764.
  68. Williams DA. Recombinant DNA advisory committee updates recommendations on gene transfer for x-linked severe combined immunodeficiency. Mol Ther. 2009 May; 17(5):751-2. PMID: 19404322.
  69. Müller LU, Williams DA. Finding the needle in the hay stack: hematopoietic stem cells in Fanconi anemia. Mutat Res. 2009 Jul 31; 668(1-2):141-9. PMID: 19508850.
    Citations: 10     Fields:    Translation:Animals
  70. Williams DA. Progress in genetic therapy for severe combined immunodeficiency associated with adenosine deaminase deficiency. Mol Ther. 2009 Apr; 17(4):577-8. PMID: 19337228.
  71. Müller LU, Daley GQ, Williams DA. Upping the ante: recent advances in direct reprogramming. Mol Ther. 2009 Jun; 17(6):947-53. PMID: 19337233.
    Citations: 30     Fields:    Translation:HumansAnimalsCells
  72. Milsom MD, Schiedlmeier B, Bailey J, Kim MO, Li D, Jansen M, Ali AM, Kirby M, Baum C, Fairbairn LJ, Williams DA. 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. PMID: 19270262.
    Citations: 16     Fields:    Translation:AnimalsCells
  73. Williams DA. New approaches in the potential treatment of HIV-acquired immunodeficiency disease. Mol Ther. 2009 Feb; 17(2):209-10. PMID: 19180109.
  74. Williams DA. ESCGT 2008: progress in clinical gene therapy. Mol Ther. 2009 Jan; 17(1):1-2. PMID: 19116631.
  75. Gan B, Sahin E, Jiang S, Sanchez-Aguilera A, Scott KL, Chin L, Williams DA, Kwiatkowski DJ, DePinho RA. 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. PMID: 19052232.
    Citations: 95     Fields:    Translation:AnimalsCells
  76. Williams DA. Sleeping beauty vector system moves toward human trials in the United States. Mol Ther. 2008 Sep; 16(9):1515-6. PMID: 18725873.
  77. Williams DA. Progress reported in two studies of clinical gene transfer into the retina. Mol Ther. 2008 Jul; 16(7):1181. PMID: 18574492.
  78. Guo F, Cancelas JA, Hildeman D, Williams DA, Zheng Y. Rac GTPase isoforms Rac1 and Rac2 play a redundant and crucial role in T-cell development. Blood. 2008 Sep 01; 112(5):1767-75. PMID: 18579797.
    Citations: 43     Fields:    Translation:AnimalsCells
  79. Sadelain M, Williams DA. A "vector drain" in US gene therapy development? Mol Ther. 2008 May; 16(5):801-2. PMID: 18432266.
  80. Müller LU, Milsom MD, Kim MO, Schambach A, Schuesler T, Williams DA. Rapid lentiviral transduction preserves the engraftment potential of Fanca(-/-) hematopoietic stem cells. Mol Ther. 2008 Jun; 16(6):1154-60. PMID: 18398427.
    Citations: 14     Fields:    Translation:HumansAnimalsCells
  81. Williams DA. Foamy virus vectors come of age. Mol Ther. 2008 Apr; 16(4):635-6. PMID: 18362918.
  82. Williams DA. NIH recombinant DNA Advisory Committee continues to ponder adverse event associated with AAV gene therapy trial. Mol Ther. 2008 Mar; 16(3):427-8. PMID: 18297091.
  83. Williams DA, Zheng Y, Cancelas JA. Rho GTPases and regulation of hematopoietic stem cell localization. Methods Enzymol. 2008; 439:365-93. PMID: 18374178.
    Citations: 30     Fields:    Translation:HumansAnimalsCells
  84. Ghiaur G, Ferkowicz MJ, Milsom MD, Bailey J, Witte D, Cancelas JA, Yoder MC, Williams DA. 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. PMID: 18083846.
    Citations: 18     Fields:    Translation:AnimalsCells
  85. Williams DA. 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. PMID: 18026181.
    Citations: 1     Fields:    Translation:Cells
  86. Williams DA. RAC reviews serious adverse event associated with AAV therapy trial. Mol Ther. 2007 Dec; 15(12):2053-4. PMID: 18026178.
  87. Thomas EK, Cancelas JA, Chae HD, Cox AD, Keller PJ, Perrotti D, Neviani P, Druker BJ, Setchell KD, Zheng Y, Harris CE, Williams DA. Rac guanosine triphosphatases represent integrating molecular therapeutic targets for BCR-ABL-induced myeloproliferative disease. Cancer Cell. 2007 Nov; 12(5):467-78. PMID: 17996650.
    Citations: 56     Fields:    Translation:HumansAnimalsCells
  88. Sherafat-Kazemzadeh R, Mehta SN, Care MM, Kim MO, Williams DA, Rose SR. Small pituitary size in children with Fanconi anemia. Pediatr Blood Cancer. 2007 Aug; 49(2):166-70. PMID: 17274008.
    Citations: 5     Fields:    Translation:Humans
  89. Williams DA. NIH decides against continuing NGVLs in their current form. Mol Ther. 2007 Jul; 15(7):1223. PMID: 17579591.
  90. Mehta PA, Ileri T, Harris RE, Williams DA, Mo J, Smolarek T, Auerbach AD, Kelly P, Davies SM. Chemotherapy for myeloid malignancy in children with Fanconi anemia. Pediatr Blood Cancer. 2007 Jun 15; 48(7):668-72. PMID: 16609946.
    Citations: 10     Fields:    Translation:Humans
  91. Milsom MD, Williams DA. 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. PMID: 17482893.
    Citations: 9     Fields:    Translation:HumansAnimalsCells
  92. Williams DA. Are lentivirus vectors safer? Mol Ther. 2007 Mar; 15(3):439. PMID: 17311027.
  93. Will E, Bailey J, Schuesler T, Modlich U, Balcik B, Burzynski B, Witte D, Layh-Schmitt G, Rudolph C, Schlegelberger B, von Kalle C, Baum C, Sorrentino BP, Wagner LM, Kelly P, Reeves L, Williams DA. 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. PMID: 17299409.
  94. Williams DA. ASGT advises NIH on funding of gene therapy trials. Mol Ther. 2007 Jan; 15(1):1-2. PMID: 17164764.
  95. Kelly PF, Radtke S, von Kalle C, Balcik B, Bohn K, Mueller R, Schuesler T, Haren M, Reeves L, Cancelas JA, Leemhuis T, Harris R, Auerbach AD, Smith FO, Davies SM, Williams DA. Stem cell collection and gene transfer in Fanconi anemia. Mol Ther. 2007 Jan; 15(1):211-9. PMID: 17164793.
    Citations: 44     Fields:    Translation:HumansCellsCTClinical Trials
  96. Williams DA. NIH funding of gene therapy trials. Mol Ther. 2006 Nov; 14(5):607. PMID: 16979942.
  97. Williams DA. Vector insertion, mutagenesis and transgene toxicity. Mol Ther. 2006 Oct; 14(4):457. PMID: 16920032.
  98. Kalfa TA, Pushkaran S, Mohandas N, Hartwig JH, Fowler VM, Johnson JF, Joiner CH, Williams DA, Zheng Y. Rac GTPases regulate the morphology and deformability of the erythrocyte cytoskeleton. Blood. 2006 Dec 01; 108(12):3637-45. PMID: 16882712.
    Citations: 42     Fields:    Translation:AnimalsCells
  99. Williams DA. A pox on your tumor. Mol Ther. 2006 Sep; 14(3):313. PMID: 16844420.
  100. Ghiaur G, Lee A, Bailey J, Cancelas JA, Zheng Y, Williams DA. Inhibition of RhoA GTPase activity enhances hematopoietic stem and progenitor cell proliferation and engraftment. Blood. 2006 Sep 15; 108(6):2087-94. PMID: 16709932.
    Citations: 25     Fields:    Translation:AnimalsCells
  101. Williams DA. Gene therapy advances but struggles to interpret safety data in small animal models. Mol Ther. 2006 Jun; 13(6):1027-8. PMID: 16684622.
  102. Williams DA, Cripe TP. Adventitious mutations in clinical grade vectors: an issue to consider? Mol Ther. 2006 May; 13(5):831-2. PMID: 16595191.
  103. Williams DA. An emerging consensus on recommendations to facilitate clinical gene transfer. Mol Ther. 2006 Apr; 13(4):637-8. PMID: 16530487.
  104. Pavitrapok C, Williams DA. 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. PMID: 16298099.
    Citations: 1     Fields:    
  105. Sudsakorn S, Kaplan L, Williams DA. 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. PMID: 16297589.
    Citations:    Fields:    
  106. Williams DA. New AAV serotypes may broaden the therapeutic pipeline to human gene therapy. Mol Ther. 2006 Jan; 13(1):1-2. PMID: 16290078.
  107. Williams DA. FDA guidance document on monitoring delayed adverse events a good first start. Mol Ther. 2005 Dec; 12(6):991-2. PMID: 16271514.
  108. Cripe TP, Thomson B, Boat TF, Williams DA. Promoting translational research in academic health centers: navigating the "roadmap". Acad Med. 2005 Nov; 80(11):1012-8. PMID: 16249299.
    Citations: 6     Fields:    Translation:Humans
  109. Schambach A, Bohne J, Chandra S, Will E, Margison GP, Williams DA, Baum C. 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. PMID: 16226060.
    Citations: 67     Fields:    Translation:HumansAnimalsCells
  110. Williams DA, Croop J, Kelly P. Gene therapy in the treatment of Fanconi anemia, a progressive bone marrow failure syndrome. Curr Opin Mol Ther. 2005 Oct; 7(5):461-6. PMID: 16248281.
    Citations: 6     Fields:    Translation:HumansCells
  111. Chandra S, Levran O, Jurickova I, Maas C, Kapur R, Schindler D, Henry R, Milton K, Batish SD, Cancelas JA, Hanenberg H, Auerbach AD, Williams DA. A rapid method for retrovirus-mediated identification of complementation groups in Fanconi anemia patients. Mol Ther. 2005 Nov; 12(5):976-84. PMID: 16084127.
    Citations: 30     Fields:    Translation:HumansCells
  112. Williams DA. A balanced decision? Regulatory reaction to the "third case". Mol Ther. 2005 Jun; 11(6):819-20. PMID: 15922952.
  113. Williams DA. The NIH roadmap: timing is everything. Mol Ther. 2005 Feb; 11(2):173. PMID: 15668128.
  114. Villella AD, Yao J, Getty RR, Juliar BE, Yiannoutsos C, Hartwell JR, Cai S, Sadat MA, Cornetta K, Williams DA, Pollok KE. Real-Time PCR: an Effective Tool for Measuring Transduction Efficiency in Human Hematopoietic Progenitor Cells. Mol Ther. 2005 Mar; 11(3):483-491. PMID: 28192683.
  115. Baum C, von Kalle C, Staal FJ, Li Z, Fehse B, Schmidt M, Weerkamp F, Karlsson S, Wagemaker G, Williams DA. Chance or necessity? Insertional mutagenesis in gene therapy and its consequences. Mol Ther. 2004 Jan; 9(1):5-13. PMID: 14741772.
  116. Neff T, Horn PA, Peterson LJ, Thomasson BM, Thompson J, Williams DA, Schmidt M, Georges GE, von Kalle C, Kiem HP. 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. PMID: 14617759.
    Citations: 29     Fields:    Translation:HumansAnimalsCells
  117. Williams DA, Baum C. Medicine. Gene therapy--new challenges ahead. Science. 2003 Oct 17; 302(5644):400-1. PMID: 14563994.
    Citations: 24     Fields:    Translation:HumansCells
  118. Chandra S, Kapur R, Chuzhanova N, Summey V, Prentice D, Barker J, Cooper DN, Williams DA. 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. PMID: 12881302.
    Citations: 1     Fields:    Translation:AnimalsCells
  119. Baum C, Düllmann J, Li Z, Fehse B, Meyer J, Williams DA, von Kalle C. Side effects of retroviral gene transfer into hematopoietic stem cells. Blood. 2003 Mar 15; 101(6):2099-114. PMID: 12511419.
    Citations: 77     Fields:    Translation:HumansAnimalsCells
  120. Williams DA, Nienhuis AW, Hawley RG, Smith FO. Gene Therapy 2000. Hematology Am Soc Hematol Educ Program. 2000; 376-393. PMID: 11701552.
    Citations:    Fields:    
  121. Pollock JD, Williams DA, Gifford MA, Li LL, Du X, Fisherman J, Orkin SH, Doerschuk CM, Dinauer MC. Mouse model of X-linked chronic granulomatous disease, an inherited defect in phagocyte superoxide production. Nat Genet. 1995 Feb; 9(2):202-9. PMID: 7719350.
    Citations: 321     Fields:    Translation:AnimalsCells
  122. Toksoz D, Williams DA. Novel human oncogene lbc detected by transfection with distinct homology regions to signal transduction products. Oncogene. 1994 Feb; 9(2):621-8. PMID: 8290273.
    Citations: 30     Fields:    Translation:HumansAnimalsCells
  123. Bodine DM, Moritz T, Donahue RE, Luskey BD, Kessler SW, Martin DI, Orkin SH, Nienhuis AW, Williams DA. 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. PMID: 7691243.
    Citations: 22     Fields:    Translation:HumansAnimalsCells
  124. Skalnik DG, Dorfman DM, Williams DA, Orkin SH. Restriction of neuroblastoma to the prostate gland in transgenic mice. Mol Cell Biol. 1991 Sep; 11(9):4518-27. PMID: 1652058.
    Citations: 7     Fields:    Translation:HumansAnimalsCells
  125. Apperley JF, Luskey BD, Williams DA. 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. PMID: 2070069.
    Citations: 14     Fields:    Translation:HumansAnimalsCells
  126. Luskey BD, Lim B, Apperley JF, Orkin SH, Williams DA. Gene transfer into murine hematopoietic stem cells and bone marrow stromal cells. Ann N Y Acad Sci. 1990; 612:398-406. PMID: 2291566.
    Citations: 1     Fields:    Translation:HumansAnimalsCells
  127. Lim B, Apperley JF, Orkin SH, Williams DA. 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. PMID: 2573068.
    Citations: 25     Fields:    Translation:HumansAnimalsCells
  128. Orkin SH, Williams DA. Gene therapy of somatic cells: status and prospects. Prog Med Genet. 1988; 7:130-42. PMID: 3277251.
    Citations:    Fields:    Translation:HumansCells
  129. Lim B, Williams DA, Orkin SH. 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. PMID: 3683389.
    Citations: 20     Fields:    Translation:HumansAnimalsCells
  130. Williams DA, Lim B, Orkin SH. Transfer and expression of human ADA in murine hematopoietic stem cells. Prog Clin Biol Res. 1987; 251:567-80. PMID: 3432278.
    Citations:    Fields:    Translation:HumansAnimalsCells
  131. Williams DA, Orkin SH, Mulligan RC. 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. PMID: 3458218.
    Citations: 48     Fields:    Translation:AnimalsCells
  132. Williams DA, Orkin SH. Somatic gene therapy. Current status and future prospects. J Clin Invest. 1986 Apr; 77(4):1053-6. PMID: 3514670.
    Citations: 11     Fields:    Translation:HumansCells
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Funded by the NIH National Center for Advancing Translational Sciences through its Clinical and Translational Science Awards Program, grant number UL1TR002541.