Gene Discovery Offers Hope for Muscular Dystrophy Treatment

In a groundbreaking study at Umeå University in Sweden, researchers have identified a specific gene that could play a crucial role in developing new treatments to prevent muscle breakdown in serious muscle diseases like muscular dystrophies. The study reveals that a gene called _fhl2b_, naturally expressed in the eye muscles, prevents them from being affected when other muscles in the body deteriorate during muscular dystrophies.

By using the Nobel Prize-winning CRISPR/Cas9 gene-editing technology, the researchers were able to express the _fhl2b_ gene in all muscles of zebrafish models with Duchenne muscular dystrophy. The results were remarkable – the zebrafish’s muscles were protected, significantly stronger, and the fish survived longer.

“You could say that the eye muscles function as both an eye-opener for understanding the disease and as a door opener to a treatment for the whole body,” says Fatima Pedrosa Domellöf, professor of eye diseases at Umeå University and one of the study’s authors.

Muscular dystrophies are a group of genetic diseases that affect muscle tissue, often leading to severe disability and reduced life expectancy. Despite intensive research, effective treatments have remained elusive for patients suffering from these conditions.

“There is a long way to go before we arrive at new treatment methods. But the results mean that we have a clear track for further research on how we can use the specific gene and protein to slow down this painful disease progression,” explains Jonas von Hofsten, associate professor at Umeå University and a researcher in the study.

While more research is needed, this discovery offers hope for potential therapies that could target the _fhl2b_ gene and its protein to combat the devastating effects of muscular dystrophies.

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