Duchenne Muscular Dystrophy: Developing a Gene Therapy Protocol for Humans
A new gene therapy for Duchenne muscular dystrophy (DMD) has shown promising results: it not only slows the progression of the disease, but also has the potential to repair damaged muscle. Human trials will begin within two years.
What is Duchenne muscular dystrophy?
DMD is a debilitating genetic disorder that disrupts the production of dystrophin, a protein that maintains muscle strength and integrity. Mobility problems typically begin in patients by age three or four and progress steadily. By adolescence, patients are unable to walk and rarely survive into their 20s.
As a genetic disorder, DMD can potentially be cured with gene therapyand scientists have even managed to achieve some success, but there is a serious obstacle. The gene encoding dystrophin is one of the largest genes and is too big to be packaged into viral vectors. These are the ones typically used to introduce healthy copies of genes into cells.
Scientists at the University of Washington have developed an alternative method that involves breaking up the protein, loading the fragments into multiple delivery vectors, and implanting instructions for reassembling the protein in muscle cells.
Practice, how can Duchenne muscular dystrophy be cured
In tests on mice with muscular dystrophy, the scientists found that large dystrophins were produced again. And the mice showed “significant physiological changes.” This stopped the disease from progressing further and even reversed some of the already atrophied muscles.
Other studies suggest an alternative approach using smaller versions of dystrophins. The first results have already been achieved in dogs. However, the team behind the new study claims that their method offers better results. The technology, with a lower dose than other types of gene therapy, leads to fewer side effects. In particular, it reduces the risk of autoimmune reactions.
Human trials are expected to begin in about two years. Moreover, the technology's potential could be applied to other genetic diseases caused by mutations in large genes.
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