DMD Exon 20 Duplication Program

The DMD Exon 20 Duplication Program is an experimental CRISPR-based gene therapy being developed by Cure Rare Disease for the treatment of Duchenne Muscular Dystrophy (DMD). This specific program is designed for patients with a duplication of exon 20 in the DMD gene, a mutation that disrupts the reading frame and prevents the production of functional dystrophin protein. The therapeutic approach utilizes CRISPR/Cas9 technology, typically delivered via an adeno-associated virus (AAV) vector, to precisely target and excise the duplicated exon. By removing the extra copy of exon 20, the therapy aims to restore the proper genetic reading frame, allowing the muscle cells to produce a functional, full-length or near-full-length dystrophin protein, thereby potentially halting or reversing the progressive muscle degeneration characteristic of DMD.

Other names

Cure Rare Disease DMD Exon 20 ProgramCRD DMD Exon 20