[NEJM] Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease

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journalㄩNew England Journal of Medicine

AuthorsㄩKiran Musunuru; Sarah A. Grandinette; Xiao Wang; Taylor R. Hudson; Kevin Briseno; Anne Marie Berry; Julia L. Hacker; Alvin Hsu; Rachel A. Silverstein; Logan T. Hille; Aysel N. Ogul; Nancy A. Robinson-Garvin; Juliana C. Small; Sarah McCague; Samantha M. Burke; Christina M. Wright; Sarah Bick; Venkata Indurthi; Shweta Sharma; Michael Jepperson; Christopher A. Vakulskas; Michael Collingwood; Katie Keogh; Ashley Jacobi; Morgan Sturgeon; Christian Brommel; Ellen Schmaljohn; Gavin Kurgan; Thomas Osborne; He Zhang; Kyle Kinney; Garrett Rettig; Christopher J. Barbosa; Sean C. Semple; Ying K. Tam; Cathleen Lutz; Lindsey A. George; Benjamin P. Kleinstiver; David R. Liu; Kim Ng; Sadik H. Kassim; Petros Giannikopoulos; Mohamad-Gabriel Alameh; Fyodor D. Urnov; Rebecca C. Ahrens-Nicklas

Published dateㄩ2025-6-12

DOIㄩ10.1056/nejmoa2504747

PDF linkㄩhttp://www.nejm.org/doi/pdf/10.1056/NEJMoa2504747

Article linkㄩhttps://doi.org/10.1056/nejmoa2504747

Article SourceㄩMassachusetts Medical Society﹝


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