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Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapy.
Chappell ME, Breda L, Tricoli L, Guerra A, Jarocha D, Castruccio Castracani C, Papp TE, Tanaka N, Hamilton N, Triebwasser MP, Ghiaccio V, Fedorky MT, Gollomp KL, Bochenek V, Roche AM, Everett JK, Cook EJ, Bushman FD, Teawtrakul N, Glentis S, Kattamis A, Mui BL, Tam YK, Weissman D, Abdulmalik O, Parhiz H, Rivella S. Chappell ME, et al. Blood. 2024 Oct 10;144(15):1633-1645. doi: 10.1182/blood.2023023349. Blood. 2024. PMID: 38949981