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Updated: Jan 16, 2026

Analyzing Satellite Cell Function During Skeletal Muscle Regeneration by Cardiotoxin Injury and Injection of Self-delivering siRNA In Vivo
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Analyzing Satellite Cell Function During Skeletal Muscle Regeneration by Cardiotoxin Injury and Injection of Self-delivering siRNA In Vivo

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Silencing Myostatin Using In Vivo Self-Assembled siRNA Protects Against Cancer- and Dexamethasone-Induced Muscle

Xin Yin1,2,3, Azhar Anwar3, Jiehao Chen3

  • 1PKU-HKUST ShenZhen-HongKong Institution, Shenzhen, Guangdong, 518057, China.

Advanced Healthcare Materials
|October 3, 2025
PubMed

Insights

This study developed a novel therapy using self-assembling siRNA to silence myostatin (MSTN), effectively preventing muscle atrophy and promoting muscle growth in mice. This offers a promising new treatment for muscle-wasting conditions.

Area of Science:

  • Biomedical Engineering
  • Molecular Biology
  • Regenerative Medicine

Background:

  • Skeletal muscle mass is vital for health, but muscle atrophy from aging, cancer, or drugs presents significant risks.
  • Current pharmacological treatments for muscle atrophy are limited, necessitating novel therapeutic strategies.

Purpose of the Study:

  • To develop and evaluate an in vivo self-assembled small interfering RNA (siRNA) therapy targeting myostatin (MSTN) to prevent muscle atrophy.
  • To investigate the efficacy of MSTN-siRNA delivered via muscle-specific peptide (MSP)-tagged small extracellular vesicles (sEVs) in promoting muscle mass and preventing atrophy.

Main Methods:

  • Designed in vivo self-assembled MSTN-siRNA constructs utilizing the liver as a scaffold for assembly.
  • Engineered MSP-tagged sEVs for selective delivery of MSTN-siRNA to muscle tissue.
  • Evaluated the therapeutic effects in healthy mice and models of cancer- and dexamethasone-induced muscle atrophy.

Main Results:

  • Successfully reduced MSTN protein levels in skeletal muscle.
  • Demonstrated promotion of muscle mass gain in healthy mice.
  • Protected skeletal muscles against atrophy in induced models, with sEV-encapsulated MSTN-siRNA proving nontoxic, nonimmunogenic, and biocompatible.

Conclusions:

  • In vivo self-assembled MSTN-siRNA delivered by MSP-tagged sEVs represents a promising therapeutic strategy for muscle atrophy.
  • This approach addresses a critical unmet need in treating muscle-wasting conditions.
  • The therapy shows potential for improving patient outcomes in various catabolic states.
Keywords:
cachexiadexamethasoneextracellular vesiclemuscle atrophymyostatinsiRNA

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