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For rescue of dogs with Duchenne muscular dystrophy (DMD)

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13.09.2018

Eric Olson, Ph.D., the founder of Exonics Therapeutics and director of Hamon Centre for Regenerative Science and Medicine UT Southwestern, has been working on the implementation of CRISPR gene editing for the treatment of Duchenne muscular dystrophy (DMD) for several years.He is a step closer in terms of reaching the goal now.

DMD is a genetic disease resulting from a mutation of the gene of dystrophin.It affects approximately 1 in 5,000 boys, causing both gradual degeneration of muscles and premature heart failure.A similar mutation is present in dogs.

Olson's team used CRISPR gene editing with regard to dog models of DMD, restoring the right level of dystrophin - a key protein in terms of the proper functioning of muscles.The level increased by as much as 92% and the research results were published in Science journal.

 "Our strategy is different from other therapeutic approaches for DMD because it edits the mutation that causes the disease and restores normal expression of the repaired dystrophin," explained Leonela Amoasii, Ph.D., the lead author of the study and assistant instructor of molecular biology in Olson's lab.

Source: fiercebiotech.com

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