Journal of translational medicine

Recent studies on gene editing with CRISPR/Cas9 for muscle-related genetic disorders

Updated

Abstract

CRISPR/Cas9 technology shows promise in treating muscle-related genetic disorders.

  • Genomic editing can potentially reverse phenotypic manifestations of various debilitating disorders.
  • CRISPR/Cas9 is being explored as a tool for gene therapy to correct specific mutations.
  • Significant advancements have been made in understanding hereditary causes of inherited myopathies and muscular dystrophies.
  • Muscular dystrophies are associated with the weakening and degeneration of skeletal muscles over time.
  • The field is experiencing progress in developing therapeutic vectors aimed at correcting genetic mutations linked to muscle diseases.
  • CRISPR/Cas9 may facilitate more effective and personalized therapies for genetic abnormalities related to muscles.

Simplified

Full Text

Full text is available at the source.

Funding

Competing interests

Declarations. Ethics approval and consent to participate: Not applicable. Consent for publication: All author consent to the publication. Competing interests: The authors declare that they have no competing interests.
PubMed

What Lands in Your Inbox Each Week:

  • 📚7 fresh studies
  • 📝plain-language summaries
  • direct links to original studies
  • 🏅top journal indicators
  • 📅weekly delivery
  • 🧘‍♂️always free