Resveratrol corrects aberrant splicing of RYR1 pre-mRNA and Ca²⁺ signal in myotonic dystrophy type 1 myotubes.

ABSTRACT: Myotonic dystrophy type 1 (DM1) is a spliceopathy related to the mis-splicing of several genes caused by sequestration of nuclear transcriptional RNA-binding factors from non-coding CUG repeats of DMPK pre-mRNAs. Dysregulation of ryanodine receptor 1 (RYR1), sarcoplasmatic/endoplasmatic Ca²⁺-ATPase (SERCA) and α1S subunit of voltage-gated Ca²⁺ channels (Ca_v1.1) is related to Ca²⁺ homeostasis and excitation-contraction coupling impairment. Though no pharmacological treatment for DM1 exists, aberrant splicing correction represents one major therapeutic target for this disease. Resveratrol (RES, 3,5,4'-trihydroxy-trans-stilbene) is a promising pharmacological tools for DM1 treatment for its ability to directly bind the DNA and RNA influencing gene e

SUBMITTER: Santoro M 

PROVIDER: S-EPMC7437583 | biostudies-literature | 2020 Sep

REPOSITORIES: biostudies-literature