Recent Progress of Antisense Oligonucleotide Therapy for

amyotrophic lateral sclerosis (ALS) antisense oligonucleotide (ASO) central nervous system (CNS) superoxide dismutase 1 (SOD1) tofersen

Journal

Genes
ISSN: 2073-4425
Titre abrégé: Genes (Basel)
Pays: Switzerland
ID NLM: 101551097

Informations de publication

Date de publication:
20 Oct 2024
Historique:
received: 28 09 2024
revised: 11 10 2024
accepted: 14 10 2024
medline: 26 10 2024
pubmed: 26 10 2024
entrez: 26 10 2024
Statut: epublish

Résumé

Amyotrophic lateral sclerosis (ALS) is a refractory neurodegenerative disease characterized by the degeneration and loss of motor neurons, typically resulting in death within five years of onset. There have been few effective treatments, making the development of robust therapies an urgent challenge. Genetic mutations have been identified as contributors to ALS, with mutations in

Identifiants

pubmed: 39457466
pii: genes15101342
doi: 10.3390/genes15101342
pii:
doi:

Substances chimiques

Superoxide Dismutase-1 EC 1.15.1.1
Oligonucleotides, Antisense 0
SOD1 protein, human 0
Oligonucleotides 0

Types de publication

Journal Article Review

Langues

eng

Sous-ensembles de citation

IM

Auteurs

Hidenori Moriyama (H)

Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada.

Toshifumi Yokota (T)

Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada.
The Friends of Garrett Cumming Research & Muscular Dystrophy Canada HM Toupin Neurological Sciences Research, Edmonton, AB T6G 2H7, Canada.

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Classifications MeSH