Therapeutic Approaches for Patients with Cystic Fibrosis Not Eligible for Current CFTR Modulators

Modulators of CFTR. Updates on clinical development and future directions

(2021) Emmanuelle Bardin et al.   EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

Mesenchymal Stem Cell exosome delivered Zinc Finger Protein activation of cystic fibrosis transmembrane conductance regulator

(2021) Olga Villamizar et al.   Journal of Extracellular Vesicles

Therapeutic promise of engineered nonsense suppressor tRNAs

(2021) Joseph J. Porter et al.   Wiley Interdisciplinary Reviews-RNA

Elexacaftor–tezacaftor–ivacaftor: The new paradigm to treat people with cystic fibrosis with at least one p.Phe508del mutation

(2021) Andrea Gramegna et al.   CURRENT OPINION IN PHARMACOLOGY

Treatment of Cystic Fibrosis: From Gene- to Cell-Based Therapies

(2021) Katelin M. Allan et al.   Frontiers in Pharmacology

Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849+10 kb C-to-T splicing mutation

(2021) Yifat S. Oren et al.   Journal of Cystic Fibrosis

New approaches to genetic therapies for cystic fibrosis

(2020) A. Christopher Boyd et al.   Journal of Cystic Fibrosis

Pharmacological approaches for targeting cystic fibrosis nonsense mutations

(2020) Jyoti Sharma et al.   EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

Antisense oligonucleotide-mediated correction of CFTR splicing improves chloride secretion in cystic fibrosis patient-derived bronchial epithelial cells

(2020) Wren E Michaels et al.   NUCLEIC ACIDS RESEARCH

Derivation of Airway Basal Stem Cells from Human Pluripotent Stem Cells

(2020) Finn J. Hawkins et al.   Cell Stem Cell

ELX-02: an investigational read-through agent for the treatment of nonsense mutation-related genetic disease

(2020) Eitan Kerem   EXPERT OPINION ON INVESTIGATIONAL DRUGS

Treating Cystic Fibrosis with mRNA and CRISPR

(2020) Alejandro Da Silva Sanchez et al.   HUMAN GENE THERAPY

New Directions in Pulmonary Gene Therapy

(2020) Amber Vu et al.   HUMAN GENE THERAPY

Ready for Repair? Gene Editing Enters the Clinic for the Treatment of Human Disease

(2020) Martijn P.T. Ernst et al.   Molecular Therapy-Methods & Clinical Development

Challenges Facing Airway Epithelial Cell-Based Therapy for Cystic Fibrosis

(2019) Andrew Berical et al.   Frontiers in Pharmacology

Rectal Organoids Enable Personalized Treatment of Cystic Fibrosis

(2019) Gitte Berkers et al.   Cell Reports

Engineered transfer RNAs for suppression of premature termination codons

(2019) John D. Lueck et al.   Nature Communications

Nonsense Mediated RNA Decay Pathway Inhibition Restores Expression and Function of W1282X CFTR

(2019) Melissa M Keenan et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Correlating CFTR Function with Clinical Features to Inform Precision Treatment of Cystic Fibrosis

(2019) Allison F McCague et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Theranostics by testing CFTR modulators in patient-derived materials: The current status and a proposal for subjects with rare CFTR mutations

(2019) Margarida D. Amaral et al.   Journal of Cystic Fibrosis

Antisense oligonucleotide eluforsen is safe and improves respiratory symptoms in F508DEL cystic fibrosis

(2019) Pavel Drevinek et al.   Journal of Cystic Fibrosis

Evaluation of eluforsen, a novel RNA oligonucleotide for restoration of CFTR function in in vitro and murine models of p.Phe508del cystic fibrosis

(2019) Wouter Beumer et al.   PLoS One

Efficient Gene Editing at Major CFTR Mutation Loci

(2019) Jinxue Ruan et al.   Molecular Therapy-Nucleic Acids

Positional effects of premature termination codons on the biochemical and biophysical properties of CFTR

(2019) Jiunn‐Tyng Yeh et al.   JOURNAL OF PHYSIOLOGY-LONDON

Functional rescue of c.3846G>A (W1282X) in patient-derived nasal cultures achieved by inhibition of nonsense mediated decay and protein modulators with complementary mechanisms of action

(2019) Onofrio Laselva et al.   Journal of Cystic Fibrosis

Comparative proteomics of respiratory exosomes in cystic fibrosis, primary ciliary dyskinesia and asthma

(2018) Virginie Rollet-Cohen et al.   Journal of Proteomics

Lipid Nanoparticle-Delivered Chemically Modified mRNA Restores Chloride Secretion in Cystic Fibrosis

(2018) Ema Robinson et al.   MOLECULAR THERAPY

Correction to: In vitro prediction of stop-codon suppression by intravenous gentamicin in patients with cystic fibrosis: a pilot study

(2018) I. Sermet-Gaudelus et al.   BMC Medicine

A revised airway epithelial hierarchy includes CFTR-expressing ionocytes

(2018) Daniel T. Montoro et al.   NATURE

Might Brushed Nasal Cells Be a Surrogate for CFTR Modulator Clinical Response?

(2018) Iwona Pranke et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Antisense oligonucleotide eluforsen improves CFTR function in F508del cystic fibrosis

(2018) Isabelle Sermet-Gaudelus et al.   Journal of Cystic Fibrosis

Cell Therapy for Cystic Fibrosis Lung Disease: Regenerative Basal Cell Amplification

(2018) Don Hayes et al.   Stem Cells Translational Medicine

Tools for translation: non-viral materials for therapeutic mRNA delivery

(2017) Khalid A. Hajj et al.   Nature Reviews Materials

Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis

(2016) Eric W F W Alton et al.   THORAX

Correction of a Cystic Fibrosis Splicing Mutation by Antisense Oligonucleotides

(2015) Susana Igreja et al.   HUMAN MUTATION

The in vitro generation of lung and airway progenitor cells from human pluripotent stem cells

(2015) Sarah X L Huang et al.   Nature Protocols

Targeted Correction and Restored Function of the CFTR Gene in Cystic Fibrosis Induced Pluripotent Stem Cells

(2015) Ana M. Crane et al.   Stem Cell Reports

Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial

(2014) Eitan Kerem et al.   Lancet Respiratory Medicine

Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients

(2013) Gerald Schwank et al.   Cell Stem Cell

mRNA as a Versatile Tool for Exogenous Protein Expression

(2012) Andreas N. Kuhn et al.   CURRENT GENE THERAPY

Statistical Analysis of Readthrough Levels for Nonsense Mutations in Mammalian Cells Reveals a Major Determinant of Response to Gentamicin

(2012) Célia Floquet et al.   PLoS Genetics

Managing Cystic Fibrosis

(2011) Malena Cohen-Cymberknoh et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

A meta-analysis of nonsense mutations causing human genetic disease

(2008) Matthew Mort et al.   HUMAN MUTATION