Development of CRISPR_SCD001, an Autologous Hematopoietic Stem Cell Gene Therapy for Sickle Cell Disease after CRISPR-Cas9 Mediated Correction

By afschroeder • September 19, 2026

DeWitt M, Campo-Fernandez B, Roy S, Kleinhenz ALW, Davila A, Lennon M, Vu JT, Krishnappa N, Said S, Tseng CH, Park SH, Bao G, Magis W, Martin DIK, Boffelli D, Wyman SK, Kohn DB, Walters MC, Romero Z. Development of CRISPR_SCD001, an Autologous Hematopoietic Stem Cell Gene Therapy for Sickle Cell Disease after CRISPR-Cas9 Mediated Correction. Mol Ther. 2026 Sep 19:S1525-0016(26)00790-2. doi: 10.1016/j.ymthe.2026.09.020. Epub ahead of print. PMID: 42760781.

Tags: DOM STAT, General Internal Medicine, Publications

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