Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease
利用患者特异性体内基因编辑技术治疗罕见遗传疾病
期刊:New England Journal of Medicine
影响因子:78.5
doi:10.1056/NEJMoa2504747
Musunuru, Kiran; Grandinette, Sarah A; Wang, Xiao; Hudson, Taylor R; Briseno, Kevin; Berry, Anne Marie; Hacker, Julia L; Hsu, Alvin; Silverstein, Rachel A; Hille, Logan T; Ogul, Aysel N; Robinson-Garvin, Nancy A; Small, Juliana C; McCague, Sarah; Burke, Samantha M; Wright, Christina M; Bick, Sarah; Indurthi, Venkata; Sharma, Shweta; Jepperson, Michael; Vakulskas, Christopher A; Collingwood, Michael; Keogh, Katie; Jacobi, Ashley; Sturgeon, Morgan; Brommel, Christian; Schmaljohn, Ellen; Kurgan, Gavin; Osborne, Thomas; Zhang, He; Kinney, Kyle; Rettig, Garrett; Barbosa, Christopher J; Semple, Sean C; Tam, Ying K; Lutz, Cathleen; George, Lindsey A; Kleinstiver, Benjamin P; Liu, David R; Ng, Kim; Kassim, Sadik H; Giannikopoulos, Petros; Alameh, Mohamad-Gabriel; Urnov, Fyodor D; Ahrens-Nicklas, Rebecca C
