Genetic “patch” showing promise

Research in Perth, WA

Researchers in Perth, WA, have developed a new kind of medicine that is offering hope for people with Duchenne Muscular Dystrophy. Different to genetic engineering, the technique works like a genetic patch, which jumps over a disease-causing part of the message as the DNA is being copied.

There is a common thread between Duchenne Muscular Dystrophy and HSP in that roughly 10% of the occurrence of each is caused by nonsense mutations. Trials of a new drug Ataluren on Duchenne cases may have relevance for treating HSPers with nonsense mutations if successful.

The Catalyst program on ABC TV covered the genetic patch recently. The program can be reviewed and the transcript is available on the ABC website.

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