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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. Among authors: fedorky mt. Blood. 2024 Oct 10;144(15):1633-1645. doi: 10.1182/blood.2023023349. Blood. 2024. PMID: 38949981
In vivo hematopoietic stem cell modification by mRNA delivery.
Breda L, Papp TE, Triebwasser MP, Yadegari A, Fedorky MT, Tanaka N, Abdulmalik O, Pavani G, Wang Y, Grupp SA, Chou ST, Ni H, Mui BL, Tam YK, Weissman D, Rivella S, Parhiz H. Breda L, et al. Among authors: fedorky mt. Science. 2023 Jul 28;381(6656):436-443. doi: 10.1126/science.ade6967. Epub 2023 Jul 27. Science. 2023. PMID: 37499029 Free PMC article.