Biblio

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Journal Article
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. 2016.
Ackermann M, Mucci A, McCabe A, Frei S, Wright K, Snapper SB, Lachmann N, Williams DA, Brendel C. Restored macrophage function ameliorates disease pathophysiology in a mouse model for IL10 receptor deficient very early onset inflammatory bowel disease. J Crohns Colitis. 2021.
Esrick EB, Lehmann LE, Biffi A, Achebe M, Brendel C, Ciuculescu MF, Daley H, MacKinnon B, Morris E, Federico A, et al. Post-Transcriptional Genetic Silencing of to Treat Sickle Cell Disease. N Engl J Med. 2020.
Labrosse R, Chu J, Armant M, Everett JK, Pellin D, Kareddy N, Frelinger AL, Henderson LA, O'Connell AE, Biswas A, et al. Outcomes of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome. Blood. 2023.
Lane SW, Williams DA, Watt FM. Modulating the stem cell niche for tissue regeneration. Nat Biotechnol. 2014.
Williams DA. The long road traveled in hematopoietic stem cell gene therapy. Mol Ther. 2022.
Brendel C, Guda S, Renella R, Bauer DE, Canver MC, Kim Y-J, Heeney MM, Klatt D, Fogel J, Milsom MD, et al. Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype. J Clin Invest. 2016.
Williams DA, Esrick E. Investigational curative gene therapy approaches to sickle cell disease. Blood Adv. 2021;5(23):5452.
Lauer A, Speroni SL, Choi M, Da X, Duncan C, McCarthy S, Krishnan V, Lusk CA, Rohde D, Hansen MBo, et al. Hematopoietic stem-cell gene therapy is associated with restored white matter microvascular function in cerebral adrenoleukodystrophy. Nat Commun. 2023;14(1):1900.
Zhang MY, Churpek JE, Keel SB, Walsh T, Lee MK, Loeb KR, Gulsuner S, Pritchard CC, Sanchez-Bonilla M, Delrow JJ, et al. Germline ETV6 mutations in familial thrombocytopenia and hematologic malignancy. Nat Genet. 2015.
Williams DA, Bennett C, Bertuch A, Bessler M, Coates T, Corey S, Dror Y, Huang J, Lipton J, Olson TS, et al. Diagnosis and treatment of pediatric acquired aplastic anemia (AAA): An initial survey of the North American Pediatric Aplastic Anemia Consortium (NAPAAC). Pediatr Blood Cancer. 2013.
Liu B, Brendel C, Vinjamur DS, Zhou Y, Harris C, McGuinness M, Manis JP, Bauer DE, Xu H, Williams DA. Development of a double shmiR lentivirus effectively targeting both BCL11A and ZNF410 for enhanced induction of fetal hemoglobin to treat β-hemoglobinopathies. Mol Ther. 2022.
L Liggett A, Cato LD, Weinstock JS, Zhang Y, S Nouraie M, Gladwin MT, Garrett ME, Ashley-Koch A, Telen M, Custer B, et al. Clonal hematopoiesis in sickle cell disease. J Clin Invest. 2022.
Zhu J, Eichler F, Biffi A, Duncan CN, Williams DA, Majzoub JA. The Changing Face of Adrenoleukodystrophy. Endocr Rev. 2020.