Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases.
Journal
EBioMedicine
ISSN: 2352-3964
Titre abrégé: EBioMedicine
Pays: Netherlands
ID NLM: 101647039
Informations de publication
Date de publication:
Jul 2019
Jul 2019
Historique:
received:
24
04
2019
revised:
31
05
2019
accepted:
18
06
2019
pubmed:
2
7
2019
medline:
18
12
2019
entrez:
2
7
2019
Statut:
ppublish
Résumé
Duchenne muscular dystrophy (DMD) is an X-linked disorder characterized by progressive muscle degeneration, caused by the absence of dystrophin. Exon skipping by antisense oligonucleotides (ASOs) has recently gained recognition as therapeutic approach in DMD. Conjugation of a peptide to the phosphorodiamidate morpholino backbone (PMO) of ASOs generated the peptide-conjugated PMOs (PPMOs) that exhibit a dramatically improved pharmacokinetic profile. When tested in animal models, PPMOs demonstrate effective exon skipping in target muscles and prolonged duration of dystrophin restoration after a treatment regime. Herein we summarize the main pathophysiological features of DMD and the emergence of PPMOs as promising exon skipping agents aiming to rescue defective gene expression in DMD and other neuromuscular diseases. The listed PPMO laboratory findings correspond to latest trends in the field and highlight the obstacles that must be overcome prior to translating the animal-based research into clinical trials tailored to the needs of patients suffering from neuromuscular diseases.
Identifiants
pubmed: 31257147
pii: S2352-3964(19)30416-5
doi: 10.1016/j.ebiom.2019.06.036
pmc: PMC6642283
pii:
doi:
Substances chimiques
Dystrophin
0
Oligonucleotides, Antisense
0
Peptides
0
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
630-645Informations de copyright
Copyright © 2019 The Authors. Published by Elsevier B.V. All rights reserved.
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