Recent Publications

Hematopoietic stem-cell gene therapy is associated with restored white matter microvascular function in cerebral adrenoleukodystrophy. Lauer A, Speroni SL, Choi M, Da X, Duncan C, McCarthy S, Krishnan V, Lusk CA, Rohde D, Hansen MB, Kalpathy-Cramer J, Loes DJ, Caruso PA, Williams DA, Mouridsen K, Emblem KE, Eichler FS, Musolino PL.Nat Commun. 2023 Apr 5;14(1):1900. doi: 10.1038/s41467-023-37262-w.PMID: 37019892 Free PMC article.

Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop. McDonald CL, Qasba P, Anderson DG, Bao G, Colvin RA, Kohn DB, Malik P, Mitchell MJ, Pu WT, Rawlings DJ, Williams DA, Flotte TR.Hum Gene Ther. 2023 Feb;34(3-4):83-89. doi: 10.1089/hum.2022.29233.tga.PMID: 36794978 No abstract available.

Secondary Neoplasms After Hematopoietic Cell Transplant for Sickle Cell Disease. Eapen M, Brazauskas R, Williams DA, Walters MC, St Martin A, Jacobs BL, Antin JH, Bona K, Chaudhury S, Coleman-Cowger VH, DiFronzo NL, Esrick EB, Field JJ, Fitzhugh CD, Kanter J, Kapoor N, Kohn DB, Krishnamurti L, London WB, Pulsipher MA, Talib S, Thompson AA, Waller EK, Wun T, Horowitz MM.J Clin Oncol. 2023 Jan 9:JCO2201203. doi: 10.1200/JCO.22.01203. Online ahead of print.PMID: 36623245

Transient neonatal hemolytic anemia due to the novel gamma globin gene mutation HBG2:C.290T>C, p.Leu97Pro (hemoglobin Wareham). Gorfinkel L, Wachter F, Luo HY, Hansbury E, Williams DA, Agarwal A, Chui DHK, Cantor AB.Pediatr Blood Cancer. 2023 Jan;70(1):e30067. doi: 10.1002/pbc.30067. Epub 2022 Oct 17.PMID: 36250986

The long road traveled in hematopoietic stem cell gene therapy. Williams DA.Mol Ther. 2022 Oct 5;30(10):3097-3099. doi: 10.1016/j.ymthe.2022.08.022. Epub 2022 Sep 13.PMID: 36099921 No abstract available.

Development of a double shmiR lentivirus effectively targeting both BCL11A and ZNF410 for enhanced induction of fetal hemoglobin to treat β-hemoglobinopathies. Liu Boya, Brendel Christian, Vinjamur Divya S., Zhou, Yu Harris Chad, McGuinness Meaghan, Manis John P., Bauer Daniel E., Xu Haiming, Williams David A. Molecular Therapy. 2022 May 6. 6:S1525-0016(22)00299-4. doi: 10.1016/j.ymthe.2022.05.002. Epub ahead of print. PMID: 35526095.

Validation of a small molecule inhibitor of PDE6D-RAS interaction with favorable anti-leukemic effects. Canovas Nunes S, De Vita S, Anighoro A, Autelitano F, Beaumont E, Klingbeil P, McGuinness M, Duvert B, Harris C, Yang L, Pokharel S, Chen C, Ermann M, Williams DA, Xu H. Blood Cancer J. 12, 64 (2022).

Clonal hematopoiesis in sickle cell disease.Liggett LA, Cato LD, Weinstock JS, Zhang Y, Nouraie SM, Gladwin MT, Garrett ME, Ashley-Koch A, Telen M, Custer B, Kelly S, Dinardo C, Sabino EC, Loureiro P, Carneiro-Proietti A, Maximo C, Reiner AP, Abecasis GR, Williams DA, Natarajan P, Bick AG, Sankaran VG.J Clin Invest. 2022 Jan 6:e156060. doi: 10.1172/JCI156060. Online ahead of print.PMID: 34990411

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.Horiguchi H, Xu H, Duvert B, Ciuculescu F, Yao Q, Sinha A, McGuinness M, Harris C, Brendel C, Troeger A, Chiarle R, Williams DA.Small GTPases. 2022 Jan;13(1):267-281. doi: 10.1080/21541248.2021.2019503.PMID: 34983288

Investigational curative gene therapy approaches to sickle cell disease.Williams DA, Esrick E.Blood Adv. 2021 Dec 14;5(23):5452. doi: 10.1182/bloodadvances.2021005567.PMID: 34905048 Review.

Congenital X-linked neutropenia with myelodysplasia and somatic tetraploidy due to a germline mutation in SEPT6.Renella R, Gagne K, Beauchamp E, Fogel J, Perlov A, Sola M, Schlaeger T, Hofmann I, Shimamura A, Ebert BL, Schmitz-Abe K, Markianos K, Murphy K, Sun L, Rockowitz S, Sliz P, Campagna DR, Springer TA, Bahl C, Agarwal S, Fleming MD, Williams DA.Am J Hematol. 2022 Jan 1;97(1):18-29. doi: 10.1002/ajh.26382. Epub 2021 Nov 3.PMID: 34677878

Mechanisms underlying genetic susceptibility to multisystem inflammatory syndrome in children (MIS-C).Chou J, Platt CD, Habiballah S, Nguyen AA, Elkins M, Weeks S, Peters Z, Day-Lewis M, Novak T, Armant M, Williams L, Rockowitz S, Sliz P, Williams DA, Randolph AG, Geha RS; Taking on COVID-19 Together Study Investigators.J Allergy Clin Immunol. 2021 Sep;148(3):732-738.e1. doi: 10.1016/j.jaci.2021.06.024. Epub 2021 Jul 2.PMID: 34224783

Evidence generation and reproducibility in cell and gene therapy research: A call to action.Abou-El-Enein M, Angelis A, Appelbaum FR, Andrews NC, Bates SE, Bierman AS, Brenner MK, Cavazzana M, Caligiuri MA, Clevers H, Cooke E, Daley GQ, Dzau VJ, Ellis LM, Fineberg HV, Goldstein LSB, Gottschalk S, Hamburg MA, Ingber DE, Kohn DB, Krainer AR, Maus MV, Marks P, Mummery CL, Pettigrew RI, Rutter JL, Teichmann SA, Terzic A, Urnov FD, Williams DA, Wolchok JD, Lawler M, Turtle CJ, Bauer G, Ioannidis JPA.Mol Ther Methods Clin Dev. 2021 Jul 21;22:11-14. doi: 10.1016/j.omtm.2021.06.012. eCollection 2021 Sep 10.PMID: 34377737

Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease.Esrick EB, Lehmann LE, Biffi A, Achebe M, Brendel C, Ciuculescu MF, Daley H, MacKinnon B, Morris E, Federico A, Abriss D, Boardman K, Khelladi R, Shaw K, Negre H, Negre O, Nikiforow S, Ritz J, Pai SY, London WB, Dansereau C, Heeney MM, Armant M, Manis JP, Williams DA.N Engl J Med. 2021 Jan 21;384(3):205-215. doi: 10.1056/NEJMoa2029392. Epub 2020 Dec 5.PMID: 33283990

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.Pulsipher MA, Lehmann LE, Bertuch AA, Sasa G, Olson T, Nakano T, Gilio A, Burroughs LM, Lipton JM, Huang JN, Dickerson K, Bertaina A, Zhuang C, Malsch M, Fleming M, Weller E, Shimamura A, Williams DA.Pediatr Blood Cancer. 2020 Oct;67(10):e28444. doi: 10.1002/pbc.28444. Epub 2020 Aug 9.PMID: 32776425 Clinical Trial.

Peripheral blood smears of children with multisystem inflammatory syndrome demonstrate prominence of early myeloid forms with morphologic evidence of toxic change.Paolino J, Williams DA.Pediatr Blood Cancer. 2021 Jan;68(1):e28551. doi: 10.1002/pbc.28551. Epub 2020 Jul 24.PMID: 32706422

Children's rare disease cohorts: an integrative research and clinical genomics initiative.Rockowitz S, LeCompte N, Carmack M, Quitadamo A, Wang L, Park M, Knight D, Sexton E, Smith L, Sheidley B, Field M, Holm IA, Brownstein CA, Agrawal PB, Kornetsky S, Poduri A, Snapper SB, Beggs AH, Yu TW, Williams DA, Sliz P.NPJ Genom Med. 2020 Jul 6;5:29. doi: 10.1038/s41525-020-0137-0. eCollection 2020.PMID: 32655885

The Changing Face of Adrenoleukodystrophy.Zhu J, Eichler F, Biffi A, Duncan CN, Williams DA, Majzoub JA.Endocr Rev. 2020 Aug 1;41(4):577-93. doi: 10.1210/endrev/bnaa013.PMID: 32364223

Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy.Brendel C, Negre O, Rothe M, Guda S, Parsons G, Harris C, McGuinness M, Abriss D, Tsytsykova A, Klatt D, Bentler M, Pellin D, Christiansen L, Schambach A, Manis J, Trebeden-Negre H, Bonner M, Esrick E, Veres G, Armant M, Williams DA.Mol Ther Methods Clin Dev. 2020 Mar 17;17:589-600. doi: 10.1016/j.omtm.2020.03.015. eCollection 2020 Jun 12.PMID: 32300607

Current and future gene therapies for hemoglobinopathies.Brendel C, Williams DA.Curr Opin Hematol. 2020 May;27(3):149-154. doi: 10.1097/MOH.0000000000000581.PMID: 32205585 Review.

Lentiviral gene therapy for X-linked chronic granulomatous disease.Kohn DB, Booth C, Kang EM, Pai SY, Shaw KL, Santilli G, Armant M, Buckland KF, Choi U, De Ravin SS, Dorsey MJ, Kuo CY, Leon-Rico D, Rivat C, Izotova N, Gilmour K, Snell K, Dip JX, Darwish J, Morris EC, Terrazas D, Wang LD, Bauser CA, Paprotka T, Kuhns DB, Gregg J, Raymond HE, Everett JK, Honnet G, Biasco L, Newburger PE, Bushman FD, Grez M, Gaspar HB, Williams DA, Malech HL, Galy A, Thrasher AJ; Net4CGD consortium.Nat Med. 2020 Feb;26(2):200-206. doi: 10.1038/s41591-019-0735-5. Epub 2020 Jan 27.PMID: 31988463 Clinical Trial.

Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study.Rogers ZR, Nakano TA, Olson TS, Bertuch AA, Wang W, Gillio A, Coates TD, Chawla A, Castillo P, Kurre P, Gamper C, Bennett CM, Joshi S, Geddis AE, Boklan J, Nalepa G, Rothman JA, Huang JN, Kupfer GM, Cada M, Glader B, Walkovich KJ, Thompson AA, Hanna R, Vlachos A, Malsch M, Weller EA, Williams DA, Shimamura A.Haematologica. 2019 Oct;104(10):1974-1983. doi: 10.3324/haematol.2018.206540. Epub 2019 Apr 4.PMID: 30948484

Highly efficient therapeutic gene editing of human hematopoietic stem cells.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.Nat Med. 2019 May;25(5):776-783. doi: 10.1038/s41591-019-0401-y. Epub 2019 Mar 25.PMID: 30911135

Pairing JAK with MEK for improved therapeutic efficiency in myeloproliferative disorders.Williams DA.J Clin Invest. 2019 Mar 4;129(4):1519-1521. doi: 10.1172/JCI127582. eCollection 2019 Mar 4.PMID: 30829649

Two Decades of Molecular Therapy and a 35-Year Personal View of Changes in Gene Therapy.Williams DA.Mol Ther. 2019 Mar 6;27(3):479-480. doi: 10.1016/j.ymthe.2019.02.003. Epub 2019 Feb 14.PMID: 30773447

Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients.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.Blood Adv. 2018 Oct 9;2(19):2505-2512. doi: 10.1182/bloodadvances.2018016725.PMID: 30282642

T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency.Clarke EL, Connell AJ, Six E, Kadry NA, Abbas AA, Hwang Y, Everett JK, Hofstaedter CE, Marsh R, Armant M, Kelsen J, Notarangelo LD, Collman RG, Hacein-Bey-Abina S, Kohn DB, Cavazzana M, Fischer A, Williams DA, Pai SY, Bushman FD.Genome Med. 2018 Sep 28;10(1):70. doi: 10.1186/s13073-018-0580-z.PMID: 30261899

DROSHA Knockout Leads to Enhancement of Viral Titers for Vectors Encoding miRNA-Adapted shRNAs.Park HH, Triboulet R, Bentler M, Guda S, Du P, Xu H, Gregory RI, Brendel C, Williams DA.Mol Ther Nucleic Acids. 2018 Sep 7;12:591-599. doi: 10.1016/j.omtn.2018.07.002. Epub 2018 Jul 9.PMID: 30195795

The pseudokinase MLKL activates PAD4-dependent NET formation in necroptotic neutrophils.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.Sci Signal. 2018 Sep 4;11(546):eaao1716. doi: 10.1126/scisignal.aao1716.PMID: 30181240

Gene Therapy for Cerebral Adrenoleukodystrophy.Williams DA, Eichler F, Duncan C.N Engl J Med. 2018 Feb 1;378(5):490-491. doi: 10.1056/NEJMc1715427.PMID: 29385368 No abstract available.

The gp130 Cytokine Interleukin-11 Regulates Engraftment of Vav1-/- Hematopoietic Stem and Progenitor Cells in Lethally Irradiated Recipients.De Vita S, Li Y, Harris CE, McGuinness MK, Ma C, Williams DA.Stem Cells. 2018 Mar;36(3):446-457. doi: 10.1002/stem.2760. Epub 2018 Jan 10.PMID: 29235178

A key role for Rac and Pak signaling in neutrophil extracellular traps (NETs) formation defines a new potential therapeutic target.Gavillet M, Martinod K, Renella R, Wagner DD, Williams DA.Am J Hematol. 2018 Feb;93(2):269-276. doi: 10.1002/ajh.24970. Epub 2017 Dec 6.PMID: 29124783

Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.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.N Engl J Med. 2017 Oct 26;377(17):1630-1638. doi: 10.1056/NEJMoa1700554. Epub 2017 Oct 4.PMID: 28976817 Clinical Trial.

Enhancement of mouse hematopoietic stem/progenitor cell function via transient gene delivery using integration-deficient lentiviral vectors.Alonso-Ferrero ME, van Til NP, Bartolovic K, Mata MF, Wagemaker G, Moulding D, Williams DA, Kinnon C, Waddington SN, Milsom MD, Howe SJ.Exp Hematol. 2018 Jan;57:21-29. doi: 10.1016/j.exphem.2017.09.003. Epub 2017 Sep 11.PMID: 28911908

The 2017 ASPHO distinguished career award goes to Holcombe E. Grier, MD.Williams DA, Orkin SH.Pediatr Blood Cancer. 2017 Jun;64 Suppl 1. doi: 10.1002/pbc.26483.PMID: 28371236 No abstract available.

167Cite Share

Evolving Gene Therapy in Primary Immunodeficiency.Thrasher AJ, Williams DA.Mol Ther. 2017 May 3;25(5):1132-1141. doi: 10.1016/j.ymthe.2017.03.018. Epub 2017 Mar 31.PMID: 28366768

Radiation-free, alternative-donor HCT for Fanconi anemia patients: results from a prospective multi-institutional study.Mehta PA, Davies SM, Leemhuis T, Myers K, Kernan NA, Prockop SE, Scaradavou A, O'Reilly RJ, Williams DA, Lehmann L, Guinan E, Margolis D, Baker KS, Lane A, Boulad F.Blood. 2017 Apr 20;129(16):2308-2315. doi: 10.1182/blood-2016-09-743112. Epub 2017 Feb 8.PMID: 28179273. Clinical Trial.

Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype.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.J Clin Invest. 2016 Oct 3;126(10):3868-3878. doi: 10.1172/JCI87885. Epub 2016 Sep 6.PMID: 27599293

RhoH participates in a multi-protein complex with the zinc finger protein kaiso that regulates both cytoskeletal structures and chemokine-induced T cells.Mino A, Troeger A, Brendel C, Cantor A, Harris C, Ciuculescu MF, Williams DA.Small GTPases. 2018 May 4;9(3):260-273. doi: 10.1080/21541248.2016.1220780. Epub 2016 Aug 31.PMID: 27574848

The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice.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.Cancer Cell. 2016 Jul 11;30(1):183. doi: 10.1016/j.ccell.2016.06.008. Epub 2016 Jul 11.PMID: 27479034 No abstract available.

Mathematical modeling of erythrocyte chimerism informs genetic intervention strategies for sickle cell disease.Altrock PM, Brendel C, Renella R, Orkin SH, Williams DA, Michor F.Am J Hematol. 2016 Sep;91(9):931-7. doi: 10.1002/ajh.24449. Epub 2016 Jul 14.PMID: 27299299

Alpharetroviral self-inactivating vectors produced by a superinfection-resistant stable packaging cell line allow genetic modification of primary human T lymphocytes.Labenski V, Suerth JD, Barczak E, Heckl D, Levy C, Bernadin O, Charpentier E, Williams DA, Fehse B, Verhoeyen E, Schambach A.Biomaterials. 2016 Aug;97:97-109. doi: 10.1016/j.biomaterials.2016.04.019. Epub 2016 Apr 26.PMID: 27162078

The Public Repository of Xenografts Enables Discovery and Randomized Phase II-like Trials in Mice.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.Cancer Cell. 2016 Apr 11;29(4):574-586. doi: 10.1016/j.ccell.2016.03.008.PMID: 27070704

Response to correspondence: Flow cytometric quantification of neutrophil extracellular traps: Limitations of the methodological approach by Ciepiela et al.Williams DA, Gavillet M.Am J Hematol. 2016 Mar;91(3):E10. doi: 10.1002/ajh.24292.PMID: 26749259

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