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Paper Details

CRISPR/Cas9-mediated excision of ALS/FTD-causing hexanucleotide repeat expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitro.
Nat Commun
23
2022
ALS, C9, C9ORF72, C9ORF72 gene, C9ORF72 haploinsufficiency, CRISPR, Cas9, FTD, HRE, RNA foci, amyotrophic lateral sclerosis, dipeptide, frontotemporal dementia, genomic locus, haploinsufficiency, hexanucleotide repeat, hexanucleotide repeat expansion, iPSC motor neurons, mouse, neurodegenerative diseases, neuronal death, patient, poly, primary cortical neurons
Author NameAffiliation
Tania F GendronMayo Clinic
Gopinath KrishnanUniversity of Massachusetts Medical School
Nils HenningerUniversity of Massachusetts Medical School
Jean-Pierre A KocherDepartment of Quantitative Health Sciences. Mayo Clinic
Leonard PetrucelliMayo Clinic
Fen-Biao GaoUniversity of Massachusetts Medical School
Erik J SontheimerRNA Therapeutics Institute and Program in Molecular Medicine, University of Massachusetts Medical School
Christian MuellerUniversity of Massachusetts Medical School
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