rAAV-CFTRΔR Rescues the Cystic Fibrosis Phenotype in Human Intestinal Organoids and Cystic Fibrosis Mice

Novel personalized therapies for cystic fibrosis: treating the basic defect in all patients

(2015) M. D. Amaral   JOURNAL OF INTERNAL MEDICINE

Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR

(2015) Claire E. Wainwright et al.   NEW ENGLAND JOURNAL OF MEDICINE

In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector

(2015) Eric Zinn et al.   Cell Reports

Evidence for Contribution of CD4+CD25+ Regulatory T Cells in Maintaining Immune Tolerance to Human Factor IX following Perinatal Adenovirus Vector Delivery

(2015) Megha S. Nivsarkar et al.   Journal of Immunology Research

Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial

(2015) Eric W F W Alton et al.   Lancet Respiratory Medicine

Biosafety of Recombinant Adeno-associated Virus Vectors

(2014) David Dismuke et al.   CURRENT GENE THERAPY

Immunological Ignorance Allows Long-Term Gene Expression After Perinatal Recombinant Adeno-Associated Virus-Mediated Gene Transfer to Murine Airways

(2014) Marianne S. Carlon et al.   HUMAN GENE THERAPY

Non-viral vectors for gene-based therapy

(2014) Hao Yin et al.   NATURE REVIEWS GENETICS

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Characterization of Defects in Ion Transport and Tissue Development in Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)-Knockout Rats

(2014) Katherine L. Tuggle et al.   PLoS One

Discovery of novel potent ΔF508-CFTR correctors that target the nucleotide binding domain

(2013) Norbert Odolczyk et al.   EMBO Molecular Medicine

Moving forward: cystic fibrosis gene therapy

(2013) U. Griesenbach et al.   HUMAN MOLECULAR GENETICS

Transcomplementation by a Truncation Mutant of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Enhances ΔF508 Processing through a Biomolecular Interaction

(2013) Liudmila Cebotaru et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

A functional CFTR assay using primary cystic fibrosis intestinal organoids

(2013) Johanna F Dekkers et al.   NATURE MEDICINE

Characterization of Nasal Potential Difference in cftr Knockout and F508del-CFTR Mice

(2013) Emilie Lyne Saussereau et al.   PLoS One

Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys

(2013) F. Mingozzi et al.   Science Translational Medicine

Absolute Determination of Single-Stranded and Self-Complementary Adeno-Associated Viral Vector Genome Titers by Droplet Digital PCR

(2013) Martin Lock et al.   Human Gene Therapy Methods

Assessment of F/HN-Pseudotyped Lentivirus as a Clinically Relevant Vector for Lung Gene Therapy

(2012) Uta Griesenbach et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

CFTR biomarkers: time for promotion to surrogate end-point

(2012) K. De Boeck et al.   EUROPEAN RESPIRATORY JOURNAL

Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue

(2012) F Mingozzi et al.   GENE THERAPY

Long-term Expansion of Epithelial Organoids From Human Colon, Adenoma, Adenocarcinoma, and Barrett's Epithelium

(2011) Toshiro Sato et al.   GASTROENTEROLOGY

New clinical diagnostic procedures for cystic fibrosis in Europe

(2011) K. De Boeck et al.   Journal of Cystic Fibrosis

Mouse models of cystic fibrosis: Phenotypic analysis and research applications

(2011) Martina Wilke et al.   Journal of Cystic Fibrosis

Sequential targeting of CFTR by BAC vectors generates a novel pig model of cystic fibrosis

(2011) N. Klymiuk et al.   JOURNAL OF MOLECULAR MEDICINE-JMM

Gene therapy finds its niche

(2011) Cormac Sheridan   NATURE BIOTECHNOLOGY

Isolation and in vitro expansion of human colonic stem cells

(2011) Peter Jung et al.   NATURE MEDICINE

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Clinical Phenotype and Genotype of Children with Borderline Sweat Test and Abnormal Nasal Epithelial Chloride Transport

(2010) Isabelle Sermet-Gaudelus et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Airway basal stem cells: a perspective on their roles in epithelial homeostasis and remodeling

(2010) J. R. Rock et al.   Disease Models & Mechanisms

Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors

(2010) Sylvie Boutin et al.   HUMAN GENE THERAPY

Disease phenotype of a ferret CFTR-knockout model of cystic fibrosis

(2010) Xingshen Sun et al.   JOURNAL OF CLINICAL INVESTIGATION

Cystic Fibrosis Transmembrane Regulator Inhibitors CFTRinh-172 and GlyH-101 Target Mitochondrial Functions, Independently of Chloride Channel Inhibition

(2010) M. Kelly et al.   JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS

Efficient Gene Transfer Into the Mouse Lung by Fetal Intratracheal Injection of rAAV2/6.2

(2010) Marianne Carlon et al.   MOLECULAR THERAPY

Toward Gene Therapy for Cystic Fibrosis Using a Lentivirus Pseudotyped With Sendai Virus Envelopes

(2010) Katsuyuki Mitomo et al.   MOLECULAR THERAPY

Paneth cells constitute the niche for Lgr5 stem cells in intestinal crypts

(2010) Toshiro Sato et al.   NATURE

Dystrophin Immunity in Duchenne's Muscular Dystrophy

(2010) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses

(2009) Roberto Calcedo et al.   JOURNAL OF INFECTIOUS DISEASES

Intersection of Gene Therapy and Progenitor Cell Biology in the Lung

(2009) Daniel J Weiss   MOLECULAR THERAPY

Dual Reporter Comparative Indexing of rAAV Pseudotyped Vectors in Chimpanzee Airway

(2009) Terence R Flotte et al.   MOLECULAR THERAPY

Ciliated epithelial cell lifespan in the mouse trachea and lung

(2008) Emma L. Rawlins et al.   AMERICAN JOURNAL OF PHYSIOLOGY-LUNG CELLULAR AND MOLECULAR PHYSIOLOGY

Cystic Fibrosis Transmembrane Regulator Missing the First Four Transmembrane Segments Increases Wild Type and ΔF508 Processing

(2008) Liudmila Cebotaru et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Disruption of the CFTR Gene Produces a Model of Cystic Fibrosis in Newborn Pigs

(2008) C. S. Rogers et al.   SCIENCE