Emerging roles of microRNAs and other non-coding transcriptome in muscular dystrophies

Area of Science:

• Biochemistry
• Genetics
• Molecular Biology

Background:

• Muscular dystrophies (MDs) are genetic neuromuscular disorders causing progressive muscle weakness.
• Aberrant genetic pathways, including dysregulated microRNAs (miRNAs) and non-coding RNAs (ncRNAs), are central to MD pathophysiology.
• In MD, ncRNAs shift from regulating physiological functions to modulating pathological mechanisms.

Purpose of the Study:

• To review the role of ncRNAs, particularly miRNAs, and their associated proteins in muscular dystrophies.
• To delineate the major ncRNAs and proteins involved in MD initiation, progression, and outcomes.
• To connect ncRNA dysregulation to core MD pathomechanisms.

Main Methods:

• Literature review of studies on ncRNAs, miRNAs, and proteins in muscular dystrophies.
• Analysis of genetic pathways and molecular mechanisms underlying MD.
• Synthesis of information across various MD subtypes.

Main Results:

• ncRNAs and miRNAs are significantly perturbed in MD, affecting gene expression.
• Key pathomechanisms in MD involving ncRNAs include myogenesis failure, structural instability, destructive processes, and signaling deficits.
• Specific miRNAs and ncRNAs, along with their protein partners, are identified as crucial players in MD.

Conclusions:

• ncRNAs and miRNAs are critical modulators of MD pathogenesis.
• Targeting ncRNA pathways presents a potential therapeutic avenue for muscular dystrophies.
• Further research into ncRNA-protein interactions is essential for understanding and treating MDs.