TCAP CRISPR gene therapy

TCAP CRISPR gene therapy is an investigational gene-editing treatment developed by Cure Rare Disease for the treatment of Limb-girdle muscular dystrophy type 2G (LGMD2G). LGMD2G is a rare, autosomal recessive muscle-wasting disease caused by mutations in the *TCAP* gene, which encodes the protein telethonin. Telethonin is a critical component of the sarcomere, the basic contractile unit of muscle fiber, and is essential for its structural integrity and assembly. This therapeutic approach utilizes CRISPR-based technology to precisely target and correct or modify pathogenic variants within the *TCAP* gene. By restoring the expression of functional telethonin protein, the therapy aims to stabilize muscle structure and halt the progression of muscle weakness. The program is currently in the preclinical stage of development.

Other names

TCAP CRISPRCRISPR-TCAPLGMD2G CRISPR therapyLGMD-2G CRISPR therapyLGMD 2G CRISPR therapy