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Article

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

4 May 2022

Abstract excerpt

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 have long been identified as a possible therapeutic for nonsense-associated diseases; however, their ability to...

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PMID: 35664697
DOI: 10.1016/j.omtn.2022.04.033

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