Antisense oligonucleotides: A primer.
Journal
Neurology. Genetics
ISSN: 2376-7839
Titre abrégé: Neurol Genet
Pays: United States
ID NLM: 101671068
Informations de publication
Date de publication:
Apr 2019
Apr 2019
Historique:
received:
17
12
2018
accepted:
14
02
2019
entrez:
24
5
2019
pubmed:
24
5
2019
medline:
24
5
2019
Statut:
epublish
Résumé
There are few disease-modifying therapeutics for neurodegenerative diseases, but successes on the development of antisense oligonucleotide (ASO) therapeutics for spinal muscular atrophy and Duchenne muscular dystrophy predict a robust future for ASOs in medicine. Indeed, existing pipelines for the development of ASO therapies for spinocerebellar ataxias, Huntington disease, Alzheimer disease, amyotrophic lateral sclerosis, Parkinson disease, and others, and increased focus by the pharmaceutical industry on ASO development, strengthen the outlook for using ASOs for neurodegenerative diseases. Perhaps the most significant advantage to ASO therapeutics over other small molecule approaches is that acquisition of the target sequence provides immediate knowledge of putative complementary oligonucleotide therapeutics. In this review, we describe the various types of ASOs, how they are used therapeutically, and the present efforts to develop new ASO therapies that will contribute to a forthcoming toolkit for treating multiple neurodegenerative diseases.
Identifiants
pubmed: 31119194
doi: 10.1212/NXG.0000000000000323
pii: NG2018009795
pmc: PMC6501637
doi:
Types de publication
Journal Article
Review
Langues
eng
Pagination
e323Subventions
Organisme : NINDS NIH HHS
ID : R01 NS097903
Pays : United States
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