Blocking myostatin shows benefits in a mouse model of OPMD

Published Date
Beatriz Bustillo Ramirez

Myostatin is an important protein produced naturally in our bodies. It stops our muscles from growing too big, in order to keep their size and strength within healthy limits.

Researchers worldwide are looking at whether blocking myostatin could make muscles grow bigger and stronger in people with neuromuscular conditions. A team at Royal Holloway University of London, led by MDUK-funded researchers Linda Popplewell and George Dickson, has recently published a study testing this approach in a mouse model of oculopharyngeal muscular dystrophy (OPMD).

In the study, the scientists used a “monoclonal antibody”. The antibody has the ability to recognise myostatin, attach to it and block its activity.

Mice with symptoms of OPMD were treated with the antibody for ten weeks. After this period, the researchers found a number of positive effects. Muscle mass and muscle fibre size had increased. The treated mice were also stronger than those who had not received the antibody.

OPMD is caused by a genetic fault in a gene called PABPN1. This produces a mutant protein that accumulates and forms clumps inside muscle cells. These clumps are thought to interfere with muscle function and have a key role in the progression of OPMD.

The researchers found that blocking myostatin did not have any effect on the amount of clumps in the muscles. This demonstrates that the treatment does not need to target the genetic cause of the condition in order to have beneficial effects. Based on these results, the scientists believe that blocking myostatin is a promising therapeutic option for OPMD that is worth exploring further.

Dr Pradeep Harish, one of the lead authors of the study, said:

Inhibition of myostatin has the potential to be used as a generic therapy for muscle-wasting conditions, either independently or in combination with other therapeutic strategies. While this method does not correct the underlying cause of the disease itself, it may be beneficial in order to stabilise the muscle and improve quality of life of patients. We are excited by future prospects.

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The scientific paper was published on the Journal of Cachexia, Sarcopenia and Muscle.

If you have any questions about this news story or any other OPMD research, please contact the MDUK Research Line on 020 7803 4813 or email

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