RNA editing technique treats severe form of muscular dystrophy
Monday, October 12, 2015 - 20:00
in Health & Medicine
An RNA editing technique called 'exon skipping' has shown preliminary success in treating a rare and severe form of muscular dystrophy that currently has no treatment. The discovery stems from the persistence of a father, whose two sons were diagnosed with a rare and severe form of muscular dystrophy, and his search for and partnership with the genetic scientist who studies the disease. The therapy is being developed with the goal of clinical trials.