Genetic Approaches for the Treatment of Facioscapulohumeral Muscular Dystrophy.

CRISPR DNA aptamers DNA decoys DUX4 RNAi antisense oligonucleotides facioscapulohumeral muscular dystrophy gene modulation

Journal

Frontiers in pharmacology

ISSN: 1663-9812

Titre abrégé: Front Pharmacol

Pays: Switzerland

ID NLM: 101548923

Informations de publication

Date de publication:
2021

Historique:

received: 16 12 2020

accepted: 01 02 2021

entrez: 29 3 2021

pubmed: 30 3 2021

medline: 30 3 2021

Statut: epublish

Résumé

Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant disorder characterized by progressive, asymmetric muscle weakness at the face, shoulders, and upper limbs, which spreads to the lower body with age. It is the third most common inherited muscular disorder worldwide. Around 20% of patients are wheelchair-bound, and some present with extramuscular manifestations. FSHD is caused by aberrant expression of the

Identifiants

DOI: 10.3389/fphar.2021.642858 PMID: 33776777 PMC: PMC7996372

pubmed: 33776777

doi: 10.3389/fphar.2021.642858

pii: 642858

pmc: PMC7996372

doi:

Types de publication

Journal Article Review

Langues

eng

Pagination

642858

Informations de copyright

Déclaration de conflit d'intérêts

TY is a co-founder and shareholder of OligomicsTx Inc., which aims to commercialize antisense technology. The remaining author declares that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

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