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

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

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...

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Bibliographic Details
Main Authors: Wooree Ko, Joseph J. Porter, Matthew T. Sipple, Katherine M. Edwards, John D. Lueck
Format: Article
Language:English
Published: Elsevier 2022-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: Oligonucleotides: Therapies and Applications
nonsense suppressor tRNA
cystic fibrosis
CFTR
nonsense mutation
premature termination codon
Online Access:http://www.sciencedirect.com/science/article/pii/S216225312200110X

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