British and French researchers have taken a key step towards developing effective gene therapy for Duchenne muscular dystrophy, it has been announced.
The treatment has successfully been used on Golden Retriever dogs who developed the disease naturally, the researchers report.
The treatment, called microdystrophin, uses a shortened version of the dystrophin gene.
Although the gene for the disease was first identified 30 years ago – one of the first disease genes to be found – it has defeated efforts to use the knowledge for an effective gene therapy, partly because of its size.
The research has been undertaken at Royal Holloway, University of London, together with the Inserm research centre in Nantes, France, and Genethon, a research organisation backed by the annual French Telethon.
The findings have been reported in Nature Communications.
Researcher Caroline Le Guiner said: “This pre-clinical study demonstrates the safety and efficacy of microdystrophin, and makes it possible to consider developing a clinical trial in patients. Indeed, this is the first time that it has been possible to treat the whole body of a large-sized animal with this protein.
"Moreover, this innovative approach allows treatment of all patients with Duchenne muscular dystrophy, regardless of the genetic mutation responsible."
Fellow researcher Professor George Dickson said: “This is tremendously exciting progress towards a gene therapy for Duchenne muscular dystrophy. The studies in Golden Retriever muscular dystrophy dogs have been spectacular and exceeded our expectations."
Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy Nature Communications 25 July 2017
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