Expansions of simple tandem repeats are responsible for almost 50 human diseases, the
majority of which are severe, degenerative, and not currently treatable or preventable. In this …

Highlights•Twenty candidate drugs are in current preclinical and clinical phases in
DM1.•Three new interventional first-in-human clinical trials got underway during 2021 …

Abstract Myotonic dystrophy type 1 (DM1) is a multisystemic neuromuscular genetic disease
with an estimated prevalence of approximately 1 million individuals based on its vast ethnic …

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

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

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 …

The CRISPR-Cas system has undoubtedly revolutionized the genome editing field, enabling
targeted gene disruption, regulation, and recovery in a guide RNA-specific manner. In this …

The human neuron-specific gene, GPM6B (Glycoprotein membrane 6B), is considered a key
gene in neural cell functionality. This gene contains an exceptionally long and strictly …

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 …