Genetic therapy could cure 1,800 diseases

New animal research into PTC124 at the University of Massachusetts Medical School indicated that it is effective in correcting the genetic disorder that causes Duchenne muscular dystrophy in about 15 percent of cases. Researchers say that DMD is sometimes triggered when the assembly process required to make proteins necessary for good health is interfered with by a DNA "nonsense mutation." The therapy works by prompting cells to ignore the mutations. The therapy, which is being developed by PTC Therapeutics, is now in human trials for DMD and cystic fibrosis. Researchers, though, have been excited by the potential for PTC124 to work against up to 1800 genetic diseases also linked to the same cellular process that triggers DMD. Those diseases include Hunter syndrome and beta thalassaemia. PTC Therapeutics recently pulled a planned IPO, saying that a series of partnerships and research grants provided enough capital for now to push its development programs without a public offering.

- see this summary on PTC124
- read the report from the Herald Sun

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