Could CRISPR help 70,000 Britons living with muscular dystrophy?

Scientists experimenting with an influential new kind of gene-editing technology known as CRISPR are becoming increasingly interested in whether it could prove game-changing for conditions such as muscular dystrophy.

Currently, it is estimated that there are around 70,000 people living in the UK who are affected by muscular dystrophy and similar conditions, which are characterised by symptoms such as muscle weakness, stiffness and pain.

In most cases, it is young boys that are affected by these muscle-wasting conditions, with approximately 100 British boys affected by Duchenne muscular dystrophy each year, for example.

However, recent research into the potential of CRISPR suggests that innovative gene-editing technology might be the answer.

By working with beagle puppies suffering from muscular dystrophy, leading scientists have been able to use CRISPR to repair genetic mutations within genomes.

They have achieved this by forcibly changing DNA sequences via injecting the puppies with viruses carrying the CRISPR gene-editing tool – which has proven successful in improving levels of dystrophin in dogs’ muscles.

“It was really gratifying to see the efficiency with which dystrophin was restored after only eight weeks,” said Eric Olson, one of the study’s co-authors.

It is hoped that the research, which was carried out by the University of Texas Southwestern Medical Centre in Dallas, could eventually pave the way for using CRISPR in humans in a similar way.

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