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. Suppressor transfer RNAs (sup-tRNAs) have the potential to treat the ∼15% of patients with DMD and nonsense mutations. We have e...