The clustered regularly interspaced short palindromic repeats (CRISPR) renaissance was
catalysed by the discovery that RNA-guided prokaryotic CRISPR-associated (Cas) proteins …

Muscular dystrophies are debilitating disorders that result in progressive weakness and
degeneration of skeletal muscle. Although the genetic mutations and clinical abnormalities …

Alternative cleavage and polyadenylation (APA) is a widespread mechanism to generate
mRNA isoforms with alternative 3′ untranslated regions (UTRs). The expression of …

Myotonic dystrophy type I (DM1) is a multisystemic autosomal-dominant inherited human
disorder that is caused by CTG microsatellite repeat expansions (MREs) in the 3 …

Clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 gene editing is
an emerging therapeutic modality that shows promise in Huntington's disease and …

Myotonic dystrophy type 1 (DM1) is the most common muscular dystrophy affecting many
different body tissues, predominantly skeletal and cardiac muscles and the central nervous …

Skeletal muscle atrophy is one of the clinical symptoms of myotonic dystrophy type 1 (DM1).
A decline in skeletal muscle regeneration is an important contributor to muscle atrophy …

Abstract Myotonic dystrophy type 1 (DM1) is caused by expanded CTG repeats (CTGexp) in
the dystrophia myotonica protein kinase (DMPK) gene, and the transcription products …

CAG/CTG repeat expansions cause over 13 neurological diseases that remain without a
cure. Because longer tracts cause more severe phenotypes, contracting them may provide a …

Myotonic Dystrophies type 1 (DM1) and type 2 (DM2) are complex multisystem diseases
without disease-based therapies. These disorders are caused by the expansions of unstable …