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Zinc finger nuclease-mediated gene editing in hematopoietic stem cells results in reactivation of fetal hemoglobin in sickle cell disease.
Lessard S, Rimmelé P, Ling H, Moran K, Vieira B, Lin YD, Rajani GM, Hong V, Reik A, Boismenu R, Hsu B, Chen M, Cockroft BM, Uchida N, Tisdale J, Alavi A, Krishnamurti L, Abedi M, Galeon I, Reiner D, Wang L, Ramezi A, Rendo P, Walters MC, Levasseur D, Peters R, Harris T, Hicks A. Lessard S, et al. Among authors: moran k. Sci Rep. 2024 Oct 16;14(1):24298. doi: 10.1038/s41598-024-74716-7. Sci Rep. 2024. PMID: 39414860 Free PMC article. Clinical Trial.
530 results