Associated Papers
RFFL-mediated protein quality control limits functional rescue of TRID-CFTR modulator combination therapy for cystic fibrosis nonsense mutations.
Tateishi H et al.
Cellular and Molecular Life Sciences : Cmls, 2026
CRISPR for cystic fibrosis: Advances and insights from a systematic review.
Nicosia L et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy, 2025
ACE-tRNAs are a platform technology for suppressing nonsense mutations that cause cystic fibrosis.
Ko W et al.
Nucleic Acids Research, 2025
Adenine base editing of CFTR using receptor targeted nanoparticles restores function to G542X cystic fibrosis airway epithelial cells.
Rose I et al.
Cellular and Molecular Life Sciences : Cmls, 2025
Adenine base editing with engineered virus-like particles rescues the CFTR mutation G542X in patient-derived intestinal organoids.
Nicosia L et al.
Iscience, 2025
Optimization of ACE-tRNAs function in translation for suppression of nonsense mutations.
Porter JJ et al.
Nucleic Acids Research, 2024
TLN468 changes the pattern of tRNA used to read through premature termination codons in CFTR.
Karri S et al.
Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society, 2024
Readthrough-induced misincorporated amino acid ratios guide mutant-specific therapeutic approaches for two CFTR nonsense mutations.
Premchandar A et al.
Frontiers in Pharmacology, 2024
The synthetic aminoglycoside ELX-02 induces readthrough of G550X-CFTR producing superfunctional protein that can be further enhanced by CFTR modulators.
Chen J et al.
American Journal of Physiology. Lung Cellular and Molecular Physiology, 2023
Targeted pseudouridylation: An approach for suppressing nonsense mutations in disease genes.
Adachi H et al.
Molecular Cell, 2023
Pharmacological Responses of the G542X-CFTR to CFTR Modulators.
Fang X et al.
Frontiers in Molecular Biosciences, 2022
Comprehensive Analysis of Combinatorial Pharmacological Treatments to Correct Nonsense Mutations in the CFTR Gene.
Venturini A et al.
International Journal of Molecular Sciences, 2021
Positional effects of premature termination codons on the biochemical and biophysical properties of CFTR.
Yeh JT et al.
The Journal of Physiology, 2020
Factors influencing readthrough therapy for frequent cystic fibrosis premature termination codons.
Pranke I et al.
Erj Open Research, 2018
Identification of the amino acids inserted during suppression of CFTR nonsense mutations and determination of their functional consequences.
Xue X et al.
Human Molecular Genetics, 2017