A synthetic molecule developed at the University of Rochester in New York was used to restore the healthy operation of muscle cells and reduce symptoms of the most common form of muscular dystrophy.
Myotonic dystrophy is triggered by a genetic mutation that creates toxic RNA that prevents proteins from doing the work that's intended. But the molecule, an antisense morpholino oligonucleotide, broke up the toxic deposits and restored cellular activity in mice.
"This study establishes a proof of concept that could be followed to develop a successful treatment for myotonic dystrophy," said neurologist Dr Charles Thornton, who led the research. "It also demonstrates the potential to reverse established symptoms of the disease after they have developed, as opposed to simply preventing them from getting worse."
- read the story from the BBC