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 …

Biomolecular condensates organize cellular functions in the absence of membranes. These
membraneless organelles can form through liquid–liquid phase separation coalescing RNA …

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 …

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 …

Technology for crosslinking and immunoprecipitation (CLIP) followed by sequencing (CLIP-
seq) has identified the transcriptomic targets of hundreds of RNA-binding proteins in cells …

RNA-binding proteins (RBPs) have emerged as important players in multiple biological
processes including transcription regulation, splicing, R-loop homeostasis, DNA …

RNA binding proteins (RBPs) act as critical facilitators of spatially regulated gene
expression. Muscleblind-like (MBNL) proteins, implicated in myotonic dystrophy and cancer …

CRISPR-Cas13 RNA endonucleases show promise for programmable RNA knockdown.
However, sequence-specific binding of Cas13 unleashes non-specific bystander RNA …

Muscleblind-like proteins (MBNLs) are RNA-binding proteins essential for the
developmental regulation of various processes including alternative splicing. Their activity is …

Historically, cellular models have been used as a tool to study myotonic dystrophy type 1
(DM1) and the validation of therapies in said pathology. However, there is a need for in vitro …