Allele-specific silencing of a dominant SETX mutation in familial amyotrophic lateral sclerosis type 4

Allele-specific silencing of a dominant SETX mutation in familial amyotrophic lateral sclerosis type 4

Summary: Amyotrophic lateral sclerosis 4 (ALS4) is an autosomal dominant motor neuron disease that is molecularly characterized by reduced R-loop levels and caused by pathogenic variants in senataxin (SETX). SETX encodes an RNA/DNA helicase that resolves three-stranded nucleic acid structures called...

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Bibliographic Details
Main Authors: Audrey Winkelsas, Athena Apfel, Brian Johnson, George Harmison, Kimberly Diaz Perez, Dongjun Li, Vivian G. Cheung, Christopher Grunseich
Format: Article
Language:English
Published: Elsevier 2025-07-01
Series:HGG Advances
Subjects:
siRNA
senataxin
amyotrophic lateral sclerosis
motor neuron disease
Online Access:http://www.sciencedirect.com/science/article/pii/S2666247725000387
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