DNA gene editing: Cure for muscular dystrophy (in mice)

Humans next, after mouse genes edited to fix deadly genetic disease. DNA editors injected into muscle correct muscle structure & make strong again.

 TECHNOLOGY & MEDICINE

DNA gene editing: Cure for muscular dystrophy (in mice)

01-01-2016
AFP News Agency

Researchers have succeeded for the first time in reversing a deadly degenerative genetic disease in mice, Duchenne muscular dystrophy (DMD), using a revolutionary new gene-editing technique.

One day soon, the same techniques could cure similar diseases in humans. 

DMD is a genetic disease that affects one in every 5,000 baby boys.

DNA EDITING TO FIX MUTATIONS

The novel gene editing technique CRISPR was used to fix gene mutations that cause the genetic disease by making precise changes to the DNA of the ailing subject, using non-pathogenic viruses as delivery vehicles for the modified genes.

Charles Gersbach, associate professor of biomedical engineering at Duke University and the leading scientist on one of the studies, in his research recently began using the new revolutionary technology of CRISPR/Cas9.

The new bioengineering technology is essentially a modified version of a bacterial defense system that targets and slices apart the DNA of invading viruses, but is now used widely by scientists to edit genes that is slice genes up and put them back together in different ways.

HALTING PROGRESSION OF DMD

Duchenne muscular dystrophy (DMD) causes progressive weakness because of genetic mutations that interfere with the production of dystrophin, a protein needed to form healthy muscle.

The research idea was to use the CRISPR gene-editing tool to correct a mutation in muscle DNA that prevents the production of the protein dystrophin.

Even though the disease DMD is incurable, dystrophin gene activity would be restored to a level to achieve adequate muscle function in a patient with the disease.

Deleting a small piece of scrambled DNA whose presence prevents the gene working normally would halted the progression of the disease leading to partial recovery (see Guardian article here).

DELIVERED DIRECTLY TO LEG MUSCLE, STRENGTHENS & CORRECTS

After refining the technique, Gersbach and his team first delivered the therapy directly to a leg muscle by injection in an adult mouse, resulting in an increase in muscle strength as some correction of muscles throughout the body, including in the heart.

"There is still a significant amount of work to do to translate this to a human therapy and demonstrate safety," Gersbach cautioned.

"But these results coming from our first experiments are very exciting."

BREAKTHROUGH STUDIES PUBLISHED IN SCIENCE

Three studies published recently in the academic journal Science said researchers employed a technique known as CRISPR to cure an adult mouse disease model suffering DMD.

"This marks the first time that CRISPR has successfully treated a genetic disease inside a fully developed living mammal with a strategy that has the potential to be translated to human therapy [to cure diseases in humans]," researchers said.

http://www.bbc.com/news/health-35205954

http://www.theguardian.com/science/2015/dec/31/breakthrough-offers-hope-to-those-with-duchenne-muscular-dystrophy

http://www.newvision.co.ug/new_vision/news/1414200/gene-editing-technique-heals-mice-muscular-dystrophy

https://en.wikipedia.org/wiki/Muscular_dystrophy

สามารถฝึกอ่านออกเสียงและดูคำแปลได้ที่ : http://www.bangkokpost.com/learning/learning-from-news/820344/dna-gene-editing-cure-for-muscular-dystrophy-in-mice