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
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