A high-fidelity CRISPR-Cas13 system improves abnormalities associated with C9ORF72-linked ALS/FTD

A high-fidelity CRISPR-Cas13 system improves abnormalities associated with C9ORF72-linked ALS/FTD

Abstract An abnormal expansion of a GGGGCC (G4C2) hexanucleotide repeat in the C9ORF72 gene is the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), two debilitating neurodegenerative disorders driven in part by gain-of-function mechanisms involving...

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Main Authors: Tristan X. McCallister, Colin K. W. Lim, Mayuri Singh, Sijia Zhang, Najah S. Ahsan, William M. Terpstra, Alisha Y. Xiong, M. Alejandra Zeballos C, Jackson E. Powell, Jenny Drnevich, Yifei Kang, Thomas Gaj
Format: Article
Language:English
Published: Nature Portfolio 2025-01-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-024-55548-5
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