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Improved specificity and efficiency of in vivo adenine base editing therapies with hybrid guide RNAs.
Whittaker MN, Testa LC, Quigley A, Brooks DL, Grandinette SA, Said H, Dwivedi G, Jindal I, Volpp D, Hacker JL, Qu P, Wang JZ, Levine MA, Ahrens-Nicklas RC, Li Q, Musunuru K, Alameh MG, Peranteau WH, Wang X. Whittaker MN, et al. Among authors: hacker jl. Nat Biomed Eng. 2025 Oct 28. doi: 10.1038/s41551-025-01545-y. Online ahead of print. Nat Biomed Eng. 2025. PMID: 41152569
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease.
Musunuru K, Grandinette SA, Wang X, Hudson TR, Briseno K, Berry AM, Hacker JL, Hsu A, Silverstein RA, Hille LT, Ogul AN, Robinson-Garvin NA, Small JC, McCague S, Burke SM, Wright CM, Bick S, Indurthi V, Sharma S, Jepperson M, Vakulskas CA, Collingwood M, Keogh K, Jacobi A, Sturgeon M, Brommel C, Schmaljohn E, Kurgan G, Osborne T, Zhang H, Kinney K, Rettig G, Barbosa CJ, Semple SC, Tam YK, Lutz C, George LA, Kleinstiver BP, Liu DR, Ng K, Kassim SH, Giannikopoulos P, Alameh MG, Urnov FD, Ahrens-Nicklas RC. Musunuru K, et al. Among authors: hacker jl. N Engl J Med. 2025 Jun 12;392(22):2235-2243. doi: 10.1056/NEJMoa2504747. Epub 2025 May 15. N Engl J Med. 2025. PMID: 40373211
Effective gene therapy for metachromatic leukodystrophy achieved with minimal lentiviral genomic integrations.
Tricoli L, Sase S, Hacker JL, Pham V, Chappell M, Breda L, Hurwitz SN, Tanaka N, Castruccio Castracani C, Guerra A, Hou Z, Schlotawa L, Radhakrishnan K, Hogenauer M, Roche A, Everett J, Bushman F, Kurre P, Ahrens-Nicklas R, Adang LA, Vanderver AL, Rivella S. Tricoli L, et al. Among authors: hacker jl. Mol Ther Nucleic Acids. 2025 Jan 25;36(1):102464. doi: 10.1016/j.omtn.2025.102464. eCollection 2025 Mar 11. Mol Ther Nucleic Acids. 2025. PMID: 40171445 Free PMC article.
543 results