Four letters. That's the difference between a suppressor tRNA that barely registers and one that restores full-length dystrophin. Nobody had ever restored full-length dystrophin in Duchenne. Not once. Until today. Our paper is out in Science Advances.
www.science.org/doi/10.1126/...
science.org
Engineering suppressor tRNAs for effective treatment of Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is a fatal disorder caused by loss of dystrophin, a protein essential for muscle cell integrity. To date, no therapeutic has restored full-length dystrophin. Suppress...
