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

8 Jan 2025

Abstract excerpt

Abstract An abnormal expansion of a GGGGCC (G 4 C 2 ) 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 transcribed forms of the repeat expansion. By utilizing a Cas13 variant with reduced collateral effects, we...

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Amyotrophic Lateral Sclerosis Research
CRISPR and Genetic Engineering
Neurogenetic and Muscular Disorders Research

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 39779681
DOI: 10.1038/s41467-024-55548-5

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A high-fidelity CRISPR-Cas13 system improves abnormalities associated with C9ORF72-linked ALS/FTDPMID 39779681Read ↗

A high-fidelity CRISPR-Cas13 system improves abnormalities associated with C9ORF72-linked ALS/FTDSemantic proximity Read ↗

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