Peer-reviewed veterinary case report
Gene therapy improves muscle function in dogs with Duchenne muscular
By Le Guiner, Caroline et al.·Published in Nature communications·2017·Atlantic Gene Therapies, France·View original on PubMed →
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Original publication title: Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy.
- Species:
- dog
Plain-English summary
A group of golden retrievers with Duchenne muscular dystrophy (DMD), a serious muscle-wasting disease, received gene therapy to help restore muscle function. The treatment involved delivering a special virus that carries a modified gene to produce a protein missing in these dogs. Over two years, the dogs showed significant improvement in muscle strength and function, with no harmful side effects reported. This promising therapy could lead to future treatments for DMD in both dogs and humans.
People also search for: golden retriever muscular dystrophy treatment · dog gene therapy for muscle disease · Duchenne muscular dystrophy in dogs
Abstract
Duchenne muscular dystrophy (DMD) is an incurable X-linked muscle-wasting disease caused by mutations in the dystrophin gene. Gene therapy using highly functional microdystrophin genes and recombinant adeno-associated virus (rAAV) vectors is an attractive strategy to treat DMD. Here we show that locoregional and systemic delivery of a rAAV2/8 vector expressing a canine microdystrophin (cMD1) is effective in restoring dystrophin expression and stabilizing clinical symptoms in studies performed on a total of 12 treated golden retriever muscular dystrophy (GRMD) dogs. Locoregional delivery induces high levels of microdystrophin expression in limb musculature and significant amelioration of histological and functional parameters. Systemic intravenous administration without immunosuppression results in significant and sustained levels of microdystrophin in skeletal muscles and reduces dystrophic symptoms for over 2 years. No toxicity or adverse immune consequences of vector administration are observed. These studies indicate safety and efficacy of systemic rAAV-cMD1 delivery in a large animal model of DMD, and pave the way towards clinical trials of rAAV-microdystrophin gene therapy in DMD patients.
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Search related cases →Original publication on PubMed: https://pubmed.ncbi.nlm.nih.gov/28742067/