CRISPR halted muscular dystrophy in dogs. Are humans next?

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ABOUT TEN YEARS ago, British veterinarians discovered an unlucky family of King Charles Spaniels whose male pups sometimes came down with a mysterious set of maladies before their first birthday. They grew clumsy and weak, and they often choked on their own tongues. To blame was a mutation on their X chromosomes, in a gene that codes for a shock-absorbing muscle protein called dystrophin. When researchers at the Royal Veterinary College realized the puppers had a canine version of the most common fatal genetic disease in children—Duchenne muscular dystrophy—they began breeding the sick spaniels with beagles to start a canine colony in the hopes of one day finding a cure.

Today, scientists report they’ve halted the progression of the disease in some of those doggy descendants using the gene editing tool known as Crispr.

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New CRISPR method takes on Duchenne Muscular Dystrophy

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The advance of CRISPR gene editing technology, which uses an RNA strand to guide an enzyme called Cas9 to cut a specific portion of DNA, has raised concerns and sparked debate as people envision a not-so-distant future populated by bioengineered super-crops, genetically flawless pets, and customized babies. While the method could be used for these purposes, it’s also showing potential as a valuable medical tool, with a seemingly new condition added each week to the list of what CRISPR may one day cure.

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Scientists a step closer to a ‘miracle’ treatment for genetic diseases

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Scientists claim they have come up with an entirely new way to rewrite the genetic code – reversing any flaws or faults.

Genetic diseases result from flaws in the body’s DNA that lead to the cells not functioning or developing as they should.  But, the genetic faults that cause diseases such as cystic fibrosis, muscular dystrophy and some cancers could be repaired in a new technique described as a “miracle of modern medicine.”

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Gene Therapy Can Improve Muscle Mass and Strength in Monkeys, Research Suggests

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Cynomolgus macaque. New research in these primates suggests that a gene delivery strategy that produces follistatin can improve muscle mass and function.

A study appearing in Science Translational Medicine puts scientists one step closer to clinical trials to test a gene delivery strategy to improve muscle mass and function in patients with certain degenerative muscle disorders.

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