Brink of a Breakthough

On Wednesday a very interesting and encouraging article was posted in The Guardian

Therapy Breakthrough

The very basic principal is to take a patients skin cells, edit them to form muscle cells and inject them back into the patient.

“Genetically corrected muscle cells or muscle progenitor cells should be able to fuse with muscle fibres,” said Akitsu Hotta, a senior author on the study, published in Stem Cell Reports. “Muscle fibres consist of hundreds or thousands of muscle cells, and even a few genetically corrected cells will be able to provide functional dystrophin protein for the entire muscle fibre. It would be very challenging to correct all the muscle fibres in the whole body, so the initial attempt should be focused on local muscles.”

Marita Pohlschmidt, director of research at the Muscular Dystrophy Campaign, said: “A number of promising potential treatments for Duchenne muscular dystrophy are currently in clinical trial. However, most of these will only result in a partially functioning dystrophin protein being produced. Using DNA enzymes to repair genetic mutations is a fast-moving research field.

“This technique has the potential to repair the genetic defect in a way that allows a fully functional protein to be made. It has the potential to develop into a treatment with a long-lasting effect, meaning children and young people may not need to be given regular treatment. However, further work and safety-testing is needed before this technology can be taken into clinical trials.”

All good stuff