New hope for treating muscular dystrophy

Thanks to CRISPR – the incredibly powerful genetic engineering process – and some bioengineers at Duke University, an adult mouse with muscular dystrophy will enter 2016 much healthier. It is also the most common and severe form of muscular dystrophy among boys, UPI reports. The disease is caused by a mutation in the gene dystrophin, located on the human X chromosome, which codes for the protein dystrophin. On an average the disordered affects around 3,600 boys, said experts.

Although the genetic cause of DMD has been known for almost 30 years, no effective treatments exist. DMD leads to break down of muscle fibers and replaces them with fatty tissues leading weak muscles. The results showed some correction of muscles throughout the body, including in the heart – a major victory because heart failure is often the cause of death for Duchenne patients. They loaded the DNA-cutting system onto a virus that infected the mice’s muscle cells, and excised from the gene a defective stretch of DNA known as an exon. “We know what genes need to be fixed for certain diseases, but getting the gene editing tools where they need to go is a huge challenge”, noted lead author Chris Nelson, Ph.D., postdoctoral fellow in Dr. Gersbach’s laboratory. Therefore, strategies would have to be developed to deliver gene-editing parts to postnatal tissues. While each delivery method had its unique benefits and improved muscle function, they found that dystrophin protein levels were highest when the treatment was injected directly into muscles.

Study’s co-researcher Dr. Leonela Amoasii from the Olson lab said, “AAV9 can efficiently infect humans in a tissue-specific manner, but it does not cause human disease or toxicity”.

GENE-EDITING injections could one day offer hope to people suffering from devastating inherited diseases, new research suggests.

“The CRISPR/Cas9 system is an adaptive immune system of single-celled organisms against invading virus”. “It simply doesn’t fit well, so we still had a packaging problem”.

The CRISPR-Cas9 gene editing system is a surprisingly simple and affordable one, discovered just three years ago and is now being examined all around the world by researchers for its promising abilities.

“This study represents a very important translational application of genome editing of DMD mutations in young mice”. Dr. Olson said that the strategy can be used in many types of mutations within the human DMD patients. However, these techniques were performed only on single-cell mammalian embryos. UTSW is one of six Wellstone Centers across the country, which work to translate scientific findings and technological developments into novel treatments for muscular dystrophy, and to promote basic, translational, and clinical research.

Researchers make progress in the fight against muscular dystrophy and new government figures show a troubling trend among heart patients.

Now, scientists at the University of Texas Southwestern, Harvard University and Duke University are using gene editing to correct a mutation that results in the disorder.

UT Southwestern’s Hamon Center for Regenerative Science and Medicine was made possible by a $10 million endowment gift from the Hamon Charitable Foundation.