Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease.

已完结 10 由 ZY 发布于 2025/5/29 8:52:56

DOI:10.1056/nejmoa2504747
作者：Kiran 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 S
文献类型：期刊论文
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New England Journal of Medicine (NEJM)

Abstract：Base editors can correct disease-causing genetic variants. After a neonate had received a diagnosis of severe carbamoyl-phosphate synthetase 1 deficiency, a disease with an estimated 50% mor

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