Article

Development of a CRISPR-Cas9-Based Cellular Model for SGCB Gene Mutation: A Platform for Investigating Gene Therapy Strategies in LGMD2E

2026-03-31

Abstract excerpt

<title>Abstract</title> Background Limb-Girdle Muscular Dystrophy Type 2E (LGMD2E) is one of the most prevalent phenotypes within the limb-girdle muscular dystrophies (LGMDs). This myopathy is caused by pathogenic mutations in the <italic>SGCB</italic> gene, which encodes β-type sarcoglycan. LGMD2E is recognized as the most prevalent sarcoglycanopathy among the Iranian population specially within the Balo...

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Literature Corpus work: a09f5e20-eb1c-58dd-8603-7951a1c66042
DOI: 10.21203/rs.3.rs-9220253/v1

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