Article

Engineered translation release factor 1 suppresses disease-causing nonsense mutations and enables multi-site nonstandard amino acids incorporation

2025-12-25

Abstract excerpt

Nonsense mutations convert sense codons into premature termination codons (PTC), resulting in early termination of translation of mRNAs, underlie ∼11% of human genetic diseases. Restoring translation of genes carrying nonsense mutations remains a major therapeutic challenge. Here we present OPENER (Overexpression of Protein-engineered eRF1 for Nonsense Elision and Readthrough), a strategy to efficiently suppress n...

Topics

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

Identifiers and source

Literature Corpus work: ca410c4f-bd76-5929-85a2-6253bad57358
DOI: 10.64898/2025.12.24.695955

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Engineered translation release factor 1 suppresses disease-causing nonsense mutations and enables multi-site nonstandard amino acids incorporationDOI 10.64898/2025.12.24.695955Read

Engineered transfer RNAs for suppression of premature termination codonsSemantic proximity Read

Mechanism-based approach in designing patient-specific combination therapies for nonsense mutation diseasesSemantic proximity Read

Introducing sense into nonsense in treatments of human genetic diseases.Semantic proximity Read

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

Drug-induced eRF1 degradation promotes readthrough and reveals a new branch of ribosome quality controlSemantic proximity Read

Single anticodon-edited tRNA therapy targeting highly prevalent Arg>Ter premature termination codons causing inherited retinal diseasesSemantic proximity Read

CRISPR-free, programmable RNA pseudouridylation to suppress premature termination codons.Semantic proximity Read

Drug-induced eRF1 degradation promotes readthrough and reveals a new branch of ribosome quality controlSemantic proximity Read

Targeting Nonsense Mutations in Diseases with Translational Read-Through-Inducing Drugs (TRIDs).Semantic proximity Read

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

Therapeutic suppression of premature termination codons: mechanisms and clinical considerations (review).Semantic proximity Read

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