New Study Shows Potential for Gene Therapy to Treat Duchenne Muscular Dystrophy
March 20, 2023
Source: drugdu
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Duchenne muscular dystrophy (DMD) is a rare and devastating genetic disorder that causes progressive muscle weakness and eventually leads to death. There is currently no cure for DMD and available treatments only offer modest benefits.
However, a recent study published in the journal Nature has shown promising results for a new approach to treating DMD using gene therapy. The researchers used a viral vector to deliver a functional copy of the dystrophin gene, which is mutated in DMD, to muscle cells in mice with the disease.
The researchers found that the gene therapy led to an improvement in muscle strength and function in the mice. They also found that the therapy was safe and well-tolerated.
The lead author of the study, Dr. Dongsheng Duan, said that the results were "very encouraging" and showed that gene therapy could be a viable option for treating DMD.
"This study demonstrates that gene therapy can be effective for treating DMD and provides a roadmap for developing gene therapies for other genetic diseases," Dr. Duan said.
Reference: https://informa.expocad.com/Exhibitor/ResourceCenter
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