Tuesday, January 5, 2016

Might this open up an avenue to cure a whole host of genetic diseases and disorders?

Members of the UT Southwestern team whose research successfully halted progression of a form of muscular dystrophy in mice included (l-r) Dr. Chengzu Long, Dr. Eric Olson, Dr. Rhonda Bassel-Duby, Dr. Leonela Amoasii, John Shelton, and Alex Mireault.

If this really results in a cure for a disease — in this case Duchenne muscular dystrophy — and not just a reduction in symptoms, the breakthrough reported by Science Daily is huge news:

Using a new gene-editing technique, a team of scientists from UT Southwestern Medical Center stopped progression of Duchenne muscular dystrophy (DMD) in young mice. … In the study published in Science, UTSW researchers used a gene-editing approach to permanently correct the DMD mutation that causes the disease in young mice.

In 2014, Dr. Eric Olson's team first used this technique -- called CRISPR/Cas9-mediated genome editing -- to correct the mutation in the germ line of mice and prevent muscular dystrophy. This paved the way for novel genome editing-based therapeutics in DMD.

Although there are not too many cases of DMD — about 1 in 5,000 boys — there are a large number of highly debilitating genetic disorders, and I wonder if the same type of approach with a CRISPR/Cas9 system might be able to find cures for all of them.

The CRISPR genome-editing technology, which was developed by a researcher at University of California at Berkeley, was picked as the "Breakthrough of the Year" scientific development by Science.

"The recent groundbreaking discoveries from the Olson laboratory using genome editing to correct the genetic mutation that causes DMD have accelerated the race to find a cure for this deadly disease," said Dr. Pradeep Mammen, Associate Professor of Internal Medicine and Co-Director of the UTSW Wellstone Center. "The challenge now lies before Wellstone Center researchers to translate these discoveries in the mouse model of DMD into a therapy for patients with DMD."



Only three days after I posted this information about possibly curing DMD with CRISPR/Cas9 genome editing and suggesting that it could be used in a similar way to cure other genetic disorders, it is being reported here that inherited blindness might also be cured by the same genome editing technique:

A research team at the Cedars-Sinai Board of Governors Regenerative Medicine Institute focused on inherited retinitis pigmentosa, a degenerative eye disease with no known cure that can lead to blindness. The researchers used a technique known as CRISPR/Cas9 to remove a genetic mutation that causes the blindness disease. CRISPR/Cas9 is adapted from a strategy used by bacteria to fight invading viruses.

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