DYSF CRISPR therapy (Cure Rare Disease)

Cure Rare Disease is developing a CRISPR-based gene editing therapy for the treatment of Limb-girdle muscular dystrophy type 2b (LGMD2b), a rare genetic disorder caused by mutations in the *DYSF* gene. The *DYSF* gene encodes dysferlin, a protein essential for the repair of skeletal muscle cell membranes. In patients with LGMD2b, the absence or deficiency of functional dysferlin leads to progressive muscle weakness and wasting. This therapeutic candidate utilizes CRISPR/Cas9 technology to precisely target and correct pathogenic variants within the *DYSF* gene or to restore the proper reading frame, thereby enabling the production of functional dysferlin protein. The program is currently in the preclinical stage, focusing on validating the efficacy and safety of the gene-editing approach in cellular and animal models.

Other names

DYSF CRISPR gene editingLGMD2B CRISPR therapyLGMD-2B CRISPR therapyLGMD 2B CRISPR therapyCure Rare Disease DYSF program