Using circular RNAs to target toxic RNA-binding proteins in amyotrophic lateral sclerosis

Using circular RNAs to target toxic RNA-binding proteins in amyotrophic lateral sclerosis

Amyotrophic lateral sclerosis (ALS) is characterized by motor neuron degeneration and is in many cases associated with mutations in genes encoding RNA-binding proteins (RBPs), including fused in sarcoma (FUS) and heterogeneous nuclear ribonuclearprotein A1 (hnRNPA1). These mutations often cause cyto...

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Bibliographic Details
Main Authors: Anne Kruse Hollensen, Matilde Helbo Sørensen, Sofie Vesterbæk Thomsen, Henriette Sylvain Thomsen, Christian Kroun Damgaard
Format: Article
Language:English
Published: Elsevier 2025-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
circRNA PROTAC
ALS
disease-causing RBPs
FUS
hnRNPA1
E3 ubiquitin ligase RC3H2
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050125001202
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