CRISPR [gene editing] halts Duchenne muscular dystrophy progression in dogs

[DocM]

Wow....

 

https://www.utsouthwestern.edu/newsroom/articles/year-2018/dmd-dogs.html

 

Quote

A study published in Science documents how scientists for the first time used gene editing to halt the progression of Duchenne muscular dystrophy (DMD) in dogs. It is seen as a major step toward a clinical trial.

 

* CRISPR gene editing technology restored muscle function in dogs to near-normal levels in the heart, diaphragm and other muscles.

* The work builds upon previous CRISPR research in which Dr. Eric Olson’s lab corrected DMD mutations in mice and human cells. 

* The technique requires only a single cut at strategic points along the subject’s DNA and is less intrusive than traditional gene-editing methods.

CRISPR halts Duchenne muscular dystrophy progression in dogs

DALLAS – Aug. 30, 2018  -- Scientists for the first time have used CRISPR gene editing to halt the progression of Duchenne muscular dystrophy (DMD) in a large mammal, according to a study by UT Southwestern that provides a strong indication that a lifesaving treatment may be in the pipeline.

The research published in Science documents unprecedented improvement in the muscle fibers of dogs with DMD – the most common fatal genetic disease in children, caused by a mutation that inhibits the production of dystrophin, a protein critical for muscle function.

Researchers used a single-cut gene-editing technique to restore dystrophin in muscle and heart tissue by up to 92 percent of normal levels. Scientists have estimated a 15 percent threshold is needed to significantly help patients.

"Children with DMD often die either because their heart loses the strength to pump, or their diaphragm becomes too weak to breathe," said Dr. Eric Olson, Director of UT Southwestern’s Hamon Center for Regenerative Science and Medicine. "This encouraging level of dystrophin expression would hopefully prevent that from happening."
>

 

[Unobscured Vision]

Cool!

[Draggendrop Veteran]

My eyes were playing games...all I saw was crispy dogs....then went....damn, hope they are all right!  /jk

 

Neat...always evolving the process.