Article

Engineered tRNA suppression of a CFTR nonsense mutation

2016-11-20

Abstract excerpt

Ten percent of human diseases are caused by ‘nonsense’ mutations that lead to premature truncation of the protein reading frame. Small molecules that promote read-through of such PTC have significant clinical promise but current iterations suffer from low in vivo efficacy and the nonselective amino acid incorporation. Alternatively, while gene-modifying approaches, such as CRISPR/Cas9, represent a long-term solut...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 1dae1413-7757-5139-bb2c-9cbbd5f0a664
DOI: 10.1101/088690

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Engineered tRNA suppression of a CFTR nonsense mutationDOI 10.1101/088690Read

Efficient suppression of endogenous CFTR nonsense mutations using anticodon-engineered transfer RNAsSemantic proximity Read

Efficient Suppression of Endogenous CFTR Nonsense Mutations Using Anticodon Engineered Transfer RNAsSemantic proximity Read

ACE-tRNAs are a platform technology for suppressing nonsense mutations that cause cystic fibrosisSemantic proximity Read

Anticodon Engineered Transfer RNAs (ACE-tRNAs) are a Platform Technology for Suppressing Nonsense MutationsSemantic proximity Read

Allele-Specific Prevention of Nonsense-Mediated Decay in Cystic Fibrosis Using Homology-Independent Genome EditingSemantic proximity Read

Optimization of Anticodon Edited Transfer RNAs (ACE-tRNAs) Function in Translation for Suppression of Nonsense MutationsSemantic proximity Read

Therapeutic promise of engineered nonsense suppressor tRNAs.Semantic proximity Read

Readthrough-induced misincorporated amino acid ratios guide mutant-specific therapeutic approaches for two CFTR nonsense mutationsSemantic proximity Read

Engineered transfer RNAs for suppression of premature termination codonsSemantic proximity Read

Anticodon Edited Transfer RNAs (ACE-tRNAs) Encoded as Therapeutic Nonviral Minimal DNA VectorsSemantic proximity Read

Identification of the amino acids inserted during suppression of CFTR nonsense mutations and determination of their functional consequences.Semantic proximity Read

Engineered mischarged transfer RNAs for correcting pathogenic missense mutations.Semantic proximity Read