UCLA research could be first step toward healing the hearts of children with Duchenne

After a progressive weakening of the muscles takes away their motor skills, and then their abilities to stand and walk, most males with Duchenne muscular dystrophy die of heart and respiratory failure in their 20s.Now, researchers at theDavid Geffen School of Medicine at UCLA are pursuing a cutting-edge way to stop heart disease in patients with Duchenne, which affects one in 5,000 male babies born in the United States. Their work, which is supported by a David Geffen School of Medicine Seed Grant, is just one of a number of projects underway at the medical school in which interdisciplinary groups of UCLA researchers are pursuing new treatments and cures.The scientists are trying to use minuscule nanoparticles to carry bits of DNA to the hearts of people with Duchenne. Inside heart cells, this new DNA would “trick” the cells’ machinery to reverse the genetic defect. The team is made up of Huan Meng, an assistant professor of nanomedicine; April Pyle, an associate professor of microbiology, immunology and molecular genetics; and Melissa Spencer, a professor of neurology.“Duchenne is such a complicated genetic disease,” Spencer said. “However, because we know the defective gene, recent advances let us directly edit the abnormal gene in muscle and heart cells in vivo.”The complex work is possible because it draws on the researchers ’ varied areas of expertise: Meng’s experience designing drug delivery systems using the latest...
Source: UCLA Newsroom: Health Sciences - Category: Universities & Medical Training Source Type: news

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(Reuters) - Sarepta Therapeutics' shares jumped 60 percent on Tuesday after promising results from a gene therapy study positioned the specialty drugmaker as a potential leader in the market for Duchenne muscular dystrophy (DMD) treatments.
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Sarepta Therapeutics on Tuesday released promising data from a small, early-stage trial of its experimental gene therapy treatment for Duchenne muscular dystrophy, showing that the drug significantly boosted an important muscle protein in three boys with the disease. Shares of Sarepta (Nasdaq: SRPT) soared Tuesday morning on the news, propelling its market cap from $6.9 billion to more than $11 billion briefly, before dropping back to $10.6 billion as of 11:15 a.m. The stock increase of 54 percent…
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(Reuters) - Sarepta Therapeutics Inc shares jumped 60 percent on Tuesday after the drugmaker reported promising results from an early-stage study testing its gene therapy in patients with Duchenne muscular dystrophy (DMD).
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(Reuters) - Sarepta Therapeutics Inc reported promising results on Tuesday from an early-stage study testing its gene therapy in patients with Duchenne muscular dystrophy (DMD), driving the company's shares up 60 percent.
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Source: Harvard Health Blog - Category: Consumer Health News Authors: Tags: Children's Health Source Type: blogs
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Source: TIME: Health - Category: Consumer Health News Authors: Tags: Uncategorized Diet/Nutrition healthytime Source Type: news
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