| 40373211 |
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
|
N Engl J Med |
2025 |
