Antisense Oligonucleotides for the Study and Treatment of ALS.
Amyotrophic lateral sclerosis
Antisense oligonucleotide
Clinical trials
Motor neuron disease
Therapy
Journal
Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics
ISSN: 1878-7479
Titre abrégé: Neurotherapeutics
Pays: United States
ID NLM: 101290381
Informations de publication
Date de publication:
07 2022
07 2022
Historique:
accepted:
02
05
2022
pubmed:
3
6
2022
medline:
26
10
2022
entrez:
2
6
2022
Statut:
ppublish
Résumé
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by motor neuron loss. ALS is now associated with mutations in numerous genes, many of which cause disease in part through toxic gain-of-function mechanisms. Antisense oligonucleotides (ASOs) are small sequences of DNA that can reduce expression of a target gene at the post-transcriptional level, making them attractive for neutralizing mutant or toxic gene products. Advancements in the medicinal chemistries of ASOs have improved their pharmacodynamic profile to allow safe and effective delivery to the central nervous system. ASO therapies for ALS have rapidly developed over the last two decades, and ASOs that target SOD1, C9orf72, FUS, and ATXN2 are now in clinical trials for familial or sporadic forms of ALS. This review discusses the current state of ASO therapies for ALS, outlining their successes from preclinical development to early clinical trials.
Identifiants
pubmed: 35653060
doi: 10.1007/s13311-022-01247-2
pii: 10.1007/s13311-022-01247-2
pmc: PMC9587169
doi:
Substances chimiques
Oligonucleotides, Antisense
0
C9orf72 Protein
0
Superoxide Dismutase-1
EC 1.15.1.1
Types de publication
Journal Article
Review
Research Support, N.I.H., Extramural
Langues
eng
Sous-ensembles de citation
IM
Pagination
1145-1158Subventions
Organisme : NIMH NIH HHS
ID : RF1 MH126723
Pays : United States
Organisme : NINDS NIH HHS
ID : R01 NS078398
Pays : United States
Informations de copyright
© 2022. The American Society for Experimental NeuroTherapeutics, Inc.
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