Antisense therapies for movement disorders.
Amyotrophic Lateral Sclerosis
/ drug therapy
Frontotemporal Dementia
/ drug therapy
Humans
Huntington Disease
/ drug therapy
Machado-Joseph Disease
/ drug therapy
Morpholinos
/ therapeutic use
Movement Disorders
/ drug therapy
Oligonucleotides, Antisense
/ therapeutic use
Parkinson Disease
/ drug therapy
Spinocerebellar Ataxias
/ drug therapy
tau Proteins
/ genetics
antisense oligonucleotide therapy
neurodegenerative and neuromuscular disorders
Journal
Movement disorders : official journal of the Movement Disorder Society
ISSN: 1531-8257
Titre abrégé: Mov Disord
Pays: United States
ID NLM: 8610688
Informations de publication
Date de publication:
08 2019
08 2019
Historique:
received:
31
12
2018
revised:
10
05
2019
accepted:
06
06
2019
pubmed:
10
7
2019
medline:
26
6
2020
entrez:
9
7
2019
Statut:
ppublish
Résumé
Currently, few disease-modifying therapies exist for degenerative movement disorders. Antisense oligonucleotides are small DNA oligonucleotides, usually encompassing ∼20 base pairs, that can potentially target any messenger RNA of interest. Antisense oligonucleotides often contain modifications to the phosphate backbone, the sugar moiety, and the nucleotide base. The development of antisense oligonucleotide therapies spinal muscular atrophy and Duchenne muscular dystrophy suggest potentially wide-ranging therapeutic applications for antisense oligonucleotides in neurology. Successes with these two diseases have heightened interest in academia and the pharmaceutical industry to develop antisense oligonucleotides for several movement disorders, including, spinocerebellar ataxias, Huntington's disease, and Parkinson's disease. Compared to small molecules, antisense oligonucleotide-based therapies have an advantage because the target disease gene sequence is the immediate path to identifying the therapeutically effective complementary antisense oligonucleotide. In this review we describe the different types of antisense oligonucleotide chemistries and their potential use for the treatment of human movement disorders. © 2019 International Parkinson and Movement Disorder Society.
Substances chimiques
Morpholinos
0
Oligonucleotides, Antisense
0
tau Proteins
0
Types de publication
Journal Article
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
1112-1119Subventions
Organisme : NINDS NIH HHS
ID : U01 NS103883
Pays : United States
Organisme : NINDS NIH HHS
ID : R37 NS033123
Pays : United States
Organisme : NINDS NIH HHS
ID : R21 NS081182
Pays : United States
Informations de copyright
© 2019 International Parkinson and Movement Disorder Society.
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