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89 results

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High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease.
Bzhilyanskaya V, Ma L, Liu S, Fox LR, Whittaker MN, Meis RJ, Choi U, Lawson A, Ma M, Theobald N, Burkett S, Sweeney CL, Lazzarotto CR, Tsai SQ, Lack JB, Wu X, Dahl GA, Malech HL, Kleinstiver BP, De Ravin SS. Bzhilyanskaya V, et al. Among authors: de ravin ss. Sci Transl Med. 2024 Oct 16;16(769):eadj6779. doi: 10.1126/scitranslmed.adj6779. Epub 2024 Oct 16. Sci Transl Med. 2024. PMID: 39413163
Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency.
Castiello MC, Brandas C, Ferrari S, Porcellini S, Sacchetti N, Canarutto D, Draghici E, Merelli I, Barcella M, Pelosi G, Vavassori V, Varesi A, Jacob A, Scala S, Basso Ricci L, Paulis M, Strina D, Di Verniere M, Sergi Sergi L, Serafini M, Holland SM, Bergerson JRE, De Ravin SS, Malech HL, Pala F, Bosticardo M, Brombin C, Cugnata F, Calzoni E, Crooks GM, Notarangelo LD, Genovese P, Naldini L, Villa A. Castiello MC, et al. Among authors: de ravin ss. Sci Transl Med. 2024 Feb 7;16(733):eadh8162. doi: 10.1126/scitranslmed.adh8162. Epub 2024 Feb 7. Sci Transl Med. 2024. PMID: 38324638 Free PMC article.
Impact of CRISPR/HDR-editing versus lentiviral transduction on long-term engraftment and clonal dynamics of HSPCs in rhesus macaques.
Lee BC, Gin A, Wu C, Singh K, Grice M, Mortlock R, Abraham D, Fan X, Zhou Y, AlJanahi A, Choi U, de Ravin SS, Shin T, Hong S, Dunbar CE. Lee BC, et al. Among authors: de ravin ss. bioRxiv [Preprint]. 2023 Dec 13:2023.12.13.571396. doi: 10.1101/2023.12.13.571396. bioRxiv. 2023. Update in: Cell Stem Cell. 2024 Apr 4;31(4):455-466.e4. doi: 10.1016/j.stem.2024.02.010 PMID: 38168153 Free PMC article. Updated. Preprint.
Small bowel disease prevalence on video capsule endoscopy in chronic granulomatous disease-associated inflammatory bowel disease.
Bhattacharya S, Wong U, Khalid M, Blaney H, Menkart MG, Oringher JL, Taneja SL, Zerbe CS, De Ravin SS, Malech HL, Holland SM, Koh C, Heller T. Bhattacharya S, et al. Among authors: de ravin ss. J Gastroenterol Hepatol. 2023 Dec;38(12):2083-2089. doi: 10.1111/jgh.16342. Epub 2023 Sep 24. J Gastroenterol Hepatol. 2023. PMID: 37743535 Free PMC article.
Intestinal microbiome and metabolome signatures in patients with chronic granulomatous disease.
Chandrasekaran P, Han Y, Zerbe CS, Heller T, DeRavin SS, Kreuzberg SA, Marciano BE, Siu Y, Jones DR, Abraham RS, Stephens MC, Tsou AM, Snapper S, Conlan S, Subramanian P, Quinones M, Grou C, Calderon V, Deming C, Leiding JW, Arnold DE, Logan BR, Griffith LM, Petrovic A, Mousallem TI, Kapoor N, Heimall JR, Barnum JL, Kapadia M, Wright N, Rayes A, Chandra S, Broglie LA, Chellapandian D, Deal CL, Grunebaum E, Lim SS, Mallhi K, Marsh RA, Murguia-Favela L, Parikh S, Touzot F, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson TR, Kang EM, Malech HL, Segre JA, Bryant CE, Holland SM, Falcone EL. Chandrasekaran P, et al. J Allergy Clin Immunol. 2023 Dec;152(6):1619-1633.e11. doi: 10.1016/j.jaci.2023.07.022. Epub 2023 Sep 1. J Allergy Clin Immunol. 2023. PMID: 37659505 Free PMC article.
The promise of in vivo HSC prime editing.
Sweeney CL, De Ravin SS. Sweeney CL, et al. Among authors: de ravin ss. Blood. 2023 Apr 27;141(17):2039-2040. doi: 10.1182/blood.2023019922. Blood. 2023. PMID: 37103948 Free article. No abstract available.
CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells.
Brault J, Liu T, Liu S, Lawson A, Choi U, Kozhushko N, Bzhilyanskaya V, Pavel-Dinu M, Meis RJ, Eckhaus MA, Burkett SS, Bosticardo M, Kleinstiver BP, Notarangelo LD, Lazzarotto CR, Tsai SQ, Wu X, Dahl GA, Porteus MH, Malech HL, De Ravin SS. Brault J, et al. Among authors: de ravin ss. Front Immunol. 2023 Jan 4;13:1067417. doi: 10.3389/fimmu.2022.1067417. eCollection 2022. Front Immunol. 2023. PMID: 36685559 Free PMC article.
Gastrointestinal and Hepatic Manifestations of Chronic Granulomatous Disease.
Yang AH, Sullivan B, Zerbe CS, De Ravin SS, Blakely AM, Quezado MM, Marciano BE, Marko J, Ling A, Kleiner DE, Gallin JI, Malech HL, Holland SM, Heller T. Yang AH, et al. Among authors: de ravin ss. J Allergy Clin Immunol Pract. 2023 May;11(5):1401-1416. doi: 10.1016/j.jaip.2022.12.039. Epub 2023 Jan 13. J Allergy Clin Immunol Pract. 2023. PMID: 36646382 Review.
89 results