Gene Editing Can Help Treat Duchenne Muscular Dystrophy

Researchers in the USA have used gene editing to treat mice with Duchenne muscular dystrophy. The researchers that achieved this impressive feat did so using the CRISPR genetic editing machine.

The paper appears on December 31, 2015 in Science. This might be a deal breaker to some since the treatment is supposed to be made for humans, but the fact that it is ethically incorrect and also the fact that mice are the mammals that share the closest resemblance to humans in various factors can also make the treatment just as effective on humans as on rats soon enough. "The best way we have to do it right now is to take advantage of viruses because they have spent billions of years evolving to figure out how to get their own viral genes into cells". Then, researchers found that the injected mice showed some restored muscle function. Causes of DMD include the presence of a defective gene in the body due to which it can not encode, dystrophin, a type of protein necessary for proper functioning of the muscle. "It's a molecular missile for gene therapy", said Dr. Leonela Amoasii, a postdoctoral researcher in the Olson lab and co-lead author of the study with Dr. Chengzu Long, Instructor of Molecular Biology. To restore expression of dystrophin proteins, Christopher Nelson et al. used the CRISPR-Cas9 gene editing system to delete exon 23, causing an additional shift in the genetic coding that allows dystrophin proteins to be expressed.

Using the controversial gene editing technology CRISPR/Cas9, researchers at the University of Texas Southwestern, Harvard University and Duke University successfully corrected a mutation that causes Duchene muscular dystrophy (DMD). Duchenne muscular dystrophy (DMD) is a debilitating genetic disease that occurs in about 1 in 3,500 males, causing muscle degeneration, loss of mobility, and premature death. But considerable work lies ahead before clinical trials can start.

Prof Adrian Thrasher, from the Institute of Child Health and Great Ormond Street Hospital, described it as an "important study demonstrating proof of principle of gene editing in vivo for neuromuscular disease, but still some way to go before translatable to human subjects". They also showed improvements to their skeletal muscle and heart. "It simply doesn't fit well, so we still had a packaging problem".

In the natural bacterial immune system, CRISPR is the mug shot that helps identify the target DNA, and Cas9 is the blade that slices the strands.

In other news NY Times reported, Duchenne muscular dystrophy is a progressive muscle-wasting disease that affects boys, putting them in wheelchairs by age 10, followed by an early death from heart failure or breathing difficulties. "But these results coming from our first experiments are very exciting".

"From here, we'll be optimising the delivery system, evaluating the approach in more severe models of DMD, and assessing efficiency and safety in larger animals with the eventual goal of getting into clinical trials".

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