FKRP CRISPR gene editing therapy

FKRP CRISPR gene editing therapy is an investigational gene editing treatment being developed by **Cure Rare Disease** for the treatment of **Limb-girdle muscular dystrophy type 2i** (LGMD2i), also known as LGMDR9. LGMD2i is a rare, progressive neuromuscular disorder caused by mutations in the **FKRP** gene, which encodes the fukutin-related protein. This protein is essential for the proper glycosylation of alpha-dystroglycan, a critical component for maintaining muscle fiber integrity. The therapy utilizes **CRISPR/Cas9** technology to directly edit and correct mutations within the endogenous FKRP gene, aiming to restore the production of functional fukutin-related protein. By addressing the underlying genetic cause at the genomic level, the therapy seeks to normalize alpha-dystroglycan glycosylation, prevent muscle degeneration, and stabilize or improve muscle function. This program is distinct from the company's gene replacement program (CRD-003) and is currently in the **preclinical** stage of development.

Other names

FKRP gene editingCRISPR-FKRPFKRP