CRISPR gene-editing gives new hope to DMD patients

An in-vitro study has given hope that CRISPR gene-editing technology may in the future be able to combat Duchenne Muscular Dystrophy, a muscle-wasting condition that affects young boys, and is nearly always fatal. Researchers announced that they had edited the genes of mice with DMD, allowing them to produce dystrophin, a protein essential for muscle function. The technique targets a variety of DMD that afflicts around 60% of sufferers, and researchers hope to swiftly move the approach forward to in-human uses. A pair of drugs to treat DMD that utilise a somewhat different "exon-skipping" approach, developed by Sarepta Therapeutics and BioMarin, are expected to be ruled upon imminently by the US FDA, and although neither is an outright cure, both have shown some promise in treating the condition in a variety of clinical trials. 

After decades of disappointingly slow progress, researchers have taken a substantial step toward a possible treatment for Duchenne muscular dystrophy with the help of a powerful new gene-editing technique.

Duchenne muscular dystrophy is a progressive muscle-wasting disease that affects boys, putting them in wheelchairs by age 10, followed by an early death from heart failure or breathing difficulties. The disease is caused by defects in a gene that encodes a protein called dystrophin, which is essential for proper muscle function.

Because the disease is devastating and incurable, and common for a hereditary illness, it has long been a target for gene therapy, though without success. An alternative treatment, drugs based on chemicals known as antisense oligonucleotides, is in clinical trials.

But gene therapy — the idea of curing a genetic disease by inserting the correct gene into damaged cells — is making a comeback. A new technique, known as Crispr-Cas9, lets researchers cut the DNA of chromosomes at selected sites to remove or insert segments.