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Gene therapy leads to long-term benefits in dog model of devastating childhood disease

Date:
June 7, 2017
Source:
Wiley
Summary:
Researchers who previously showed that a gene therapy treatment could save the lives of dogs with a deadly disease called myotubular myopathy — a type of muscular dystrophy that affects the skeletal muscles—have found that the therapy is long-lasting.
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Researchers who previously showed that a gene therapy treatment could save the lives of dogs with a deadly disease called myotubular myopathy -- a type of muscular dystrophy that affects the skeletal muscles -- have found that the therapy is long-lasting. The results support a clinical trial in patients.

Myotubular myopathy is estimated to affect 1 in 50,000 male births, and boys born with the condition often die in the first year of life.

In this latest study, investigators found that replacing the MTM1 gene, which is mutated in patients, leads to sustained preservation of muscle strength and neurologic function in dogs over 4 years.

"This regenerative technology, termed AAV gene transfer, provided long-lasting benefit to the entire musculature of affected dogs that would have otherwise perished, extending a healthy lifespan for more than 4 years," said Dr. Martin Childers, senior author of the Muscle & Nerve study and a UW Medicine researcher in Seattle.


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Materials provided by Wiley. Note: Content may be edited for style and length.


Journal Reference:

  1. Thomas Mccaffrey, Michela Guglieri, Alexander P. Murphy, Katherine Bushby, Anna Johnson, John P. Bourke. Cardiac involvement in female carriers of duchenne or becker muscular dystrophy. Muscle & Nerve, 2017; 55 (6): 810 DOI: 10.1002/mus.25445

Cite This Page:

Wiley. "Gene therapy leads to long-term benefits in dog model of devastating childhood disease." ScienceDaily. ScienceDaily, 7 June 2017. <www.sciencedaily.com/releases/2017/06/170607142157.htm>.
Wiley. (2017, June 7). Gene therapy leads to long-term benefits in dog model of devastating childhood disease. ScienceDaily. Retrieved June 2, 2025 from www.sciencedaily.com/releases/2017/06/170607142157.htm
Wiley. "Gene therapy leads to long-term benefits in dog model of devastating childhood disease." ScienceDaily. www.sciencedaily.com/releases/2017/06/170607142157.htm (accessed June 2, 2025).

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