Genetic medicines for CF: Hype versus reality

Rapid Expansion of Human Epithelial Stem Cells Suitable for Airway Tissue Engineering

(2016) Colin R. Butler et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Lentivirus pre-packed with Cas9 protein for safer gene editing

(2016) J G Choi et al.   GENE THERAPY

High-fidelity CRISPR–Cas9 nucleases with no detectable genome-wide off-target effects

(2016) Benjamin P. Kleinstiver et al.   NATURE

Enhancing homology-directed genome editing by catalytically active and inactive CRISPR-Cas9 using asymmetric donor DNA

(2016) Christopher D Richardson et al.   NATURE BIOTECHNOLOGY

Airway acidification initiates host defense abnormalities in cystic fibrosis mice

(2016) V. S. Shah et al.   SCIENCE

TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs

(2016) Shingo Suzuki et al.   Molecular Therapy-Nucleic Acids

Minicircle DNA Provides Enhanced and Prolonged Transgene Expression Following Airway Gene Transfer

(2016) Mustafa M. Munye et al.   Scientific Reports

Systematic quantification of HDR and NHEJ reveals effects of locus, nuclease, and cell type on genome-editing

(2016) Yuichiro Miyaoka et al.   Scientific Reports

A Phase I/IIa Safety and Efficacy Study of Nebulized Liposome-mediated Gene Therapy for Cystic Fibrosis Supports a Multidose Trial

(2015) Eric W. F. W. Alton et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Multiplex Genome-Edited T-cell Manufacturing Platform for “Off-the-Shelf” Adoptive T-cell Immunotherapies

(2015) Laurent Poirot et al.   CANCER RESEARCH

Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System

(2015) Bernd Zetsche et al.   CELL

Cystic Fibrosis Gene Therapy in the UK and Elsewhere

(2015) Uta Griesenbach et al.   HUMAN GENE THERAPY

Origins of Cystic Fibrosis Lung Disease

(2015)   NEW ENGLAND JOURNAL OF MEDICINE

Rationally engineered Cas9 nucleases with improved specificity

(2015) I. M. Slaymaker et al.   SCIENCE

Nanoparticles that deliver triplex-forming peptide nucleic acid molecules correct F508del CFTR in airway epithelium

(2015) Nicole Ali McNeer et al.   Nature Communications

Functional Gene Correction for Cystic Fibrosis in Lung Epithelial Cells Generated from Patient iPSCs

(2015) Amy L. Firth et al.   Cell Reports

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

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

TALEN-Mediated Generation and Genetic Correction of Disease-Specific Human Induced Pluripotent Stem Cells

(2014) Sivaprakash Ramalingam et al.   CURRENT GENE THERAPY

Understanding how cystic fibrosis mutations disrupt CFTR function: From single molecules to animal models

(2014) Yiting Wang et al.   INTERNATIONAL JOURNAL OF BIOCHEMISTRY & CELL BIOLOGY

WS1.5 Restoration of the CFTR function by antisense oligonucleotide splicing modulation

(2014) M. Irony-Tur Sinai et al.   Journal of Cystic Fibrosis

Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial

(2014) Stéphane Palfi et al.   LANCET

Overcoming the Cystic Fibrosis Sputum Barrier to Leading Adeno-associated Virus Gene Therapy Vectors

(2014) Benjamin S Schuster et al.   MOLECULAR THERAPY

Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV

(2014) Pablo Tebas 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

Targeted genome editing by lentiviral protein transduction of zinc-finger and TAL-effector nucleases

(2014) Yujia Cai et al.   eLife

Native Small Airways Secrete Bicarbonate

(2013) A. K. M. Shamsuddin et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep

(2013) Eric W.F.W. Alton et al.   BIOMATERIALS

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

(2013) Gerald Schwank et al.   Cell Stem Cell

Toxicology study assessing efficacy and safety of repeated administration of lipid/DNA complexes to mouse lung

(2013) E W F W Alton et al.   GENE THERAPY

Intestinal CFTR expression alleviates meconium ileus in cystic fibrosis pigs

(2013) David A. Stoltz et al.   JOURNAL OF CLINICAL INVESTIGATION

Modified Foxp3 mRNA protects against asthma through an IL-10–dependent mechanism

(2013) Lauren E. Mays et al.   JOURNAL OF CLINICAL INVESTIGATION

High efficiency gene transfer to airways of mice using influenza hemagglutinin pseudotyped lentiviral vectors

(2013) Manij Patel et al.   JOURNAL OF GENE MEDICINE

Defining the disease liability of variants in the cystic fibrosis transmembrane conductance regulator gene

(2013) Patrick R Sosnay et al.   NATURE GENETICS

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

(2013) A. Biffi et al.   SCIENCE

The Committee for Advanced Therapies' of the European Medicines Agency Reflection Paper on Management of Clinical Risks Deriving from Insertional Mutagenesis

(2013) Alessandro Aiuti et al.   Human Gene Therapy Clinical Development

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

Obligate Ligation-Gated Recombination (ObLiGaRe): Custom-designed nuclease-mediated targeted integration through nonhomologous end joining

(2012) M. Maresca et al.   GENOME RESEARCH

Gene Therapy for Primary Immunodeficiencies

(2012) Christine Rivat et al.   HUMAN GENE THERAPY

Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells

(2012) Maarten Holkers et al.   NUCLEIC ACIDS RESEARCH

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

Lentiviral Vector Gene Transfer to Porcine Airways

(2012) Patrick L Sinn et al.   Molecular Therapy-Nucleic Acids

Cystic Fibrosis Transmembrane Conductance Regulator Intracellular Processing, Trafficking, and Opportunities for Mutation-Specific Treatment

(2011) Mark P. Rogan et al.   CHEST

Pre-clinical evaluation of three non-viral gene transfer agents for cystic fibrosis after aerosol delivery to the ovine lung

(2011) G McLachlan et al.   GENE THERAPY

Mouse models of cystic fibrosis: Phenotypic analysis and research applications

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

In vivo genome editing restores haemostasis in a mouse model of haemophilia

(2011) Hojun Li et al.   NATURE

Expression of therapeutic proteins after delivery of chemically modified mRNA in mice

(2011) Michael S D Kormann et al.   NATURE BIOTECHNOLOGY

Generating the optimal mRNA for therapy: HPLC purification eliminates immune activation and improves translation of nucleoside-modified, protein-encoding mRNA

(2011) Katalin Karikó et al.   NUCLEIC ACIDS RESEARCH

Modularly assembled designer TAL effector nucleases for targeted gene knockout and gene replacement in eukaryotes

(2011) Ting Li et al.   NUCLEIC ACIDS RESEARCH

A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity

(2011) Claudio Mussolino et al.   NUCLEIC ACIDS RESEARCH

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

(2010) Katsuyuki Mitomo et al.   MOLECULAR THERAPY

Ion transport across CF and normal murine olfactory and ciliated epithelium

(2009) B. R. Grubb et al.   AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY

Limitations of the Murine Nose in the Development of Nonviral Airway Gene Transfer

(2009) Uta Griesenbach et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

CFTR Delivery to 25% of Surface Epithelial Cells Restores Normal Rates of Mucus Transport to Human Cystic Fibrosis Airway Epithelium

(2009) Liqun Zhang et al.   PLOS BIOLOGY

Gene transfer to the lung: Lessons learned from more than 2 decades of CF gene therapy

(2008) Uta Griesenbach et al.   ADVANCED DRUG DELIVERY REVIEWS

Validation of Nasal Potential Difference Measurements in Gut-Corrected CF Knockout Mice

(2008) Uta Griesenbach et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Lentivirus Vector Can Be Readministered to Nasal Epithelia without Blocking Immune Responses

(2008) P. L. Sinn et al.   JOURNAL OF VIROLOGY

Incorporation of Pseudouridine Into mRNA Yields Superior Nonimmunogenic Vector With Increased Translational Capacity and Biological Stability

(2008) Katalin Karikó et al.   MOLECULAR THERAPY

CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression

(2008) Stephen C Hyde et al.   NATURE BIOTECHNOLOGY