An oligonucleotide treatment for muscular dystrophy has shown promising results in pre-clinical trials. It is estimated that the new treatment could be effective for over half of muscular dystrophy patients.

Muscular dystrophy is a hereditary condition characterised by a progressive weakening and wasting of the muscles. It is caused by a malfunction in the gene that codes for dystrophin protein. Dystrophin acts as a support scaffold for the muscle cells, and without it the cells become easily damaged and unstable.

However, a new oligonucleotide treatment is being developed that aims to increase the production of dystrophin in humans to restore muscle function.

Antisense oligonucleotides are small sections of single stranded DNA that can be designed to bind to the mRNA of faulty genes. They work by silencing the gene and are proving to be a promising avenue of research for multiple genetic disorders.

A ‘cocktail’ of multiple oligonucleotides was trialled in mice engineered to have the faulty muscular dystrophy gene and human muscle cells taken from muscular dystrophy patients. In both cases, the treatment enabled the muscle cells to produce a shorter version of the dystrophin protein. The shorter dystrophin protein has been associated with symptomatic relief in muscular dystrophy patients.

The current FDA approved oligonucleotide drug for muscular dystrophy is only effective for 13% of patients, as it can only bind to a small selection of the faulty dystrophin gene variants. However, these findings indicate that by varying the oligonucleotide mixture, the drugs could prove effective in approximately 65% of muscular dystrophy patients. The researchers now aim to reduce the number of oligonucleotides in the cocktail to reduce the cost of the drug and make it more accessible to patients.