Scientists Find a Way to Edit DNA to Cure Genetic Disorders

An innovative gene-manipulation technique known as CRISPR-Cas9 has shown further promise as a treatment for Duchenne muscular dystrophy (DMD), all while avoiding potential ethical dilemmas.

A series of three animal studies was published in the journal Science on Thursday, demonstrating the effectiveness of CRISPR-Cas9 in selectively editing the defective gene responsible for DMD, which affects approximately 1 in every 5,600 to 7,700 males ages 5 through 24 in the U.S. The researchers used harmless virus (adeno-associated virus, or AAV) to inject the CRISPR-Cas9 gene editing system into living mice; once inside, it was able to snip away one of the genetic mutations thought to be responsible for DMD. Afterwards, these injected mice had a degree of their muscle function restored, in some cases for as long as six months.

“We are very encouraged by these results in animals and feel this work is an important step forward in realizing the potential of gene editing to correct disease-causing mutations in muscle tissue of patients with DMD,” says Amy Wagers, one of the many study authors and a Harvard University researcher.

http://www.goodnewsnetwork.org/using-crispr-to-cure-genetic-disorders/