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 …

Lung cancer (LC) is a heterogeneous disease consisting mainly of two subtypes, non-small
cell lung cancer (NSCLC) and small cell lung cancer (SCLC), and remains the leading …

Huntington's disease (HD) is a fatal, dominantly inherited neurodegenerative disorder
caused by CAG trinucleotide expansion in exon 1 of the huntingtin (HTT) gene. Since the …

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 …

Abstract Myotonic dystrophy type 1 (DM1) is an RNA-dominant disease whose pathogenesis
stems from the functional loss of muscleblind-like RNA-binding proteins (RBPs), which …

Clustered regularly interspaced short palindromic repeats (CRISPR)-Cas13 has drawn
broad interest to control gene expression and cell fate at the RNA level in general. Apart …

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 …

Myotonic dystrophy type 1 (DM1) is a dominant genetic disease in which the expansion of
long CTG trinucleotides in the 3′ UTR of the myotonic dystrophy protein kinase (DMPK) …

Expansion of CAG and CTG (CWG) triplet repeats causes several inherited neurological
diseases. The CWG repeat diseases are thought to involve complex pathogenic …

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 …