CRISPR/Cas9-Mediated Excision of ALS/FTD-Causing Hexanucleotide Repeat Expansion in C9ORF72 Rescues Major Disease Mechanisms in Vivo and in Vitro
Katharina E. Meijboom,Abbas Abdallah,Nicholas P. Fordham,Hiroko Nagase,Tomás Rodriguez,Carolyn Kraus,Tania F. Gendron,Gopinath Krishnan,Rustam Esanov,Nadja S. Andrade,Matthew J. Rybin,Melina Ramic,Zachary D. Stephens,Alireza Edraki,Meghan T. Blackwood,Aydan Kahriman,Nils Henninger,Jean-Pierre A. Kocher,Michael Benatar,Michael H. Brodsky,Leonard Petrucelli,Fen-Biao Gao,Erik J. Sontheimer,Robert H. Brown,Zane Zeier,Christian Mueller Nature Communications（2022）
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Amyotrophic lateral sclerosis,CRISPR-Cas9 genome editing,Science,Humanities and Social Sciences,multidisciplinary
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