China's Original RNA Editing Technology First Applied to Rare Disease Treatment in the World
The RNA editing technology LEAPER developed by Changping Laboratory and Professor Wei Wensheng's team at Peking University was used for Duchenne muscular dystrophy treatment; three children showed significant and sustained improvement in motor function after one year of LE051 treatment follow-up. The technological achievements were published simultaneously in Cell journal, opening a new track for genetic rare diseases like DMD.
Shanghai Children's Medical Center