Efficient suppression of endogenous CFTR nonsense mutations using anticodon-engineered transfer RNAs

• Prif Awduron: Wooree Ko (Awdur), Joseph J. Porter (Awdur), Matthew T. Sipple (Awdur), Katherine M. Edwards (Awdur), John D. Lueck (Awdur)
• Fformat: Llyfr
• Cyhoeddwyd: Elsevier, 2022-06-01T00:00:00Z.
• Pynciau: article
• Mynediad Ar-lein: Connect to this object online.

Nonsense mutations or premature termination codons (PTCs) comprise ∼11% of all genetic lesions, which result in over 7,000 distinct genetic diseases. Due to their outsized impact on human health, considerable effort has been made to find therapies for nonsense-associated diseases. Suppressor tRNAs h...