Article
Challenges associated with homologous directed repair using CRISPR-Cas9 and TALEN to edit the DMD genetic mutation in canine Duchenne muscular dystrophy.
PloS one - 1 Jan 2020
Mata López Sara, Balog-Alvarez Cynthia, Vitha Stanislav, Bettis Amanda K, Canessa Emily H, Kornegay Joe N, Nghiem Peter P
Abstract excerpt
Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene that abolish the expression of dystrophin protein. Dogs with the genetic homologue, golden retriever muscular dystrophy dog (GRMD), have a splice site mutation that leads to skipping of exon 7 and a stop codon in the DMD transcript. Gene editing via homology-directed repair (HDR) has been used in the mdx mouse model of DMD but not in GRMD....
Topics
- Animals
- CRISPR-Cas Systems
- Dogs
- Dystrophin
- Gene Editing
- Genetic Therapy
- Muscular Dystrophy, Duchenne
- Mutation
- Myoblasts
- Transcription Activator-Like Effector Nucleases