New developments of Crispr technology for potential cure to dystrophy

The prestigious journal Science has published an important article reporting the correction of a genetic mutation in four dogs with Duchenne’s disease – an extremely serious type of muscular dystrophy.

The team of researchers from the UT Southwestern Medical Center of  Dallas and the Royal Veterinary College of London have applied the Crispr gene editing technology to ensure that muscles produce a crucial protein, whose deficiency results in dystrophy.

Among humans, Duchenne muscular dystrophy affects at least 20,000 children and young adults in the world, worsening their living conditions and leading to death. They rarely live longer than 30 years.

Only one approved therapy currently exists for this condition–it is Exondys 51, developed by biotech Sarepta Therapeutics, which obtained approval in 2016 and is sold at $300,000 per year.

The Wall Street Journal estimates that at least 45 biotech companies are working on developing a new therapy for the disorder, and Crispr technology could be the solution many people are waiting for.  However, it is important to recall that the same technology has recently raised much criticism and skepticism, as some studies– published in Nature–have revealed that it can edit some other DNA parts  at the same time, triggering cancer.