Recombinant Adeno-Associated Virus-Mediated Editing of the G551D Cystic Fibrosis Transmembrane Conductance Regulator Mutation in Ferret Airway Basal Cells

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

(2021) Julian D. Gillmore et al.   NEW ENGLAND JOURNAL OF MEDICINE

Functional correction of CFTR mutations in human airway epithelial cells using adenine base editors

(2021) Sateesh Krishnamurthy et al.   NUCLEIC ACIDS RESEARCH

Potential of helper-dependent Adenoviral vectors in CRISPR-cas9-mediated lung gene therapy

(2021) Ranmal Avinash Bandara et al.   Cell and Bioscience

Evaluating CRISPR-based prime editing for cancer modeling and CFTR repair in organoids

(2021) Maarten H Geurts et al.   Life Science Alliance

AAV5 delivery of CRISPR-Cas9 supports effective genome editing in mouse lung airway

(2021) Shun-Qing Liang et al.   MOLECULAR THERAPY

AAV-mediated gene editing lights up the lung

(2021) Ziying Yan et al.   MOLECULAR THERAPY

Viral Vectors, Animal Models, and Cellular Targets for Gene Therapy of Cystic Fibrosis Lung Disease

(2020) Yinghua Tang et al.   HUMAN GENE THERAPY

Gene Therapy for Cystic Fibrosis Paved the Way for the Use of Adeno-Associated Virus in Gene Therapy

(2020) William B. Guggino et al.   HUMAN GENE THERAPY

Highly Efficient Gene Editing of Cystic Fibrosis Patient-Derived Airway Basal Cells Results in Functional CFTR Correction

(2020) Shingo Suzuki et al.   MOLECULAR THERAPY

Correction of Airway Stem Cells: Genome Editing Approaches for the Treatment of Cystic Fibrosis

(2020) Nicholas E King et al.   HUMAN GENE THERAPY

Detargeting Lentiviral-Mediated CFTR Expression in Airway Basal Cells Using miR-106b

(2020) Soon H. Choi et al.   Genes

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

(2020) Haydar Frangoul et al.   NEW ENGLAND JOURNAL OF MEDICINE

TALEN-Mediated Gene Targeting for Cystic Fibrosis-Gene Therapy

(2019) Emily Xia et al.   Genes

Methodologies for Improving HDR Efficiency

(2019) Mingjie Liu et al.   Frontiers in Genetics

Highly Efficient Transgenesis in Ferrets Using CRISPR/Cas9-Mediated Homology-Independent Insertion at the ROSA26 Locus

(2019) Miao Yu et al.   Scientific Reports

In vitro validation of a CRISPR-mediated CFTR correction strategy for preclinical translation in pigs

(2019) Zhichang Peter Zhou et al.   HUMAN GENE THERAPY

Advances in gene therapy for cystic fibrosis lung disease

(2019) Ziying Yan et al.   HUMAN MOLECULAR GENETICS

Stimulation of CRISPR-mediated homology-directed repair by an engineered RAD18 variant

(2019) Tarun S. Nambiar et al.   Nature Communications

Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

(2019) Peter G. Middleton et al.   NEW ENGLAND JOURNAL OF MEDICINE

High-Efficiency, Selection-free Gene Repair in Airway Stem Cells from Cystic Fibrosis Patients Rescues CFTR Function in Differentiated Epithelia

(2019) Sriram Vaidyanathan et al.   Cell Stem Cell

Systematic genome editing of the genes on zebrafish Chromosome 1 by CRISPR/Cas9

(2019) Yonghua Sun et al.   GENOME RESEARCH

Transducing Airway Basal Cells with a Helper-Dependent Adenoviral Vector for Lung Gene Therapy

(2018) Huibi Cao et al.   HUMAN GENE THERAPY

CFTR modulator theratyping: Current status, gaps and future directions

(2018) John Paul Clancy et al.   Journal of Cystic Fibrosis

GC content elevates mutation and recombination rates in the yeastSaccharomyces cerevisiae

(2018) Denis A. Kiktev et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A single-cell atlas of the airway epithelium reveals the CFTR-rich pulmonary ionocyte

(2018) Lindsey W. Plasschaert et al.   NATURE

A revised airway epithelial hierarchy includes CFTR-expressing ionocytes

(2018) Daniel T. Montoro et al.   NATURE

Bicarbonate in cystic fibrosis

(2017) Karl Kunzelmann et al.   Journal of Cystic Fibrosis

Inhibition of 53BP1 favors homology-dependent DNA repair and increases CRISPR–Cas9 genome-editing efficiency

(2017) Marella D Canny et al.   NATURE BIOTECHNOLOGY

Ectopic expression of RAD52 and dn53BP1 improves homology-directed repair during CRISPR–Cas9 genome editing

(2017) Bruna S. Paulsen et al.   Nature Biomedical Engineering

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

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

RS-1 enhances CRISPR/Cas9- and TALEN-mediated knock-in efficiency

(2016) Jun Song et al.   Nature Communications

Increasing the efficiency of precise genome editing with CRISPR-Cas9 by inhibition of nonhomologous end joining

(2015) Takeshi Maruyama et al.   NATURE BIOTECHNOLOGY

Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing in mammalian cells

(2015) Van Trung Chu et al.   NATURE BIOTECHNOLOGY

Therapeutic genome editing: prospects and challenges

(2015) David Benjamin Turitz Cox et al.   NATURE MEDICINE

Origins of Cystic Fibrosis Lung Disease

(2015) David A. Stoltz et al.   NEW ENGLAND JOURNAL OF MEDICINE

Nuclear domain ‘knock-in’ screen for the evaluation and identification of small molecule enhancers of CRISPR-based genome editing

(2015) Jordan Pinder et al.   NUCLEIC ACIDS RESEARCH

Ferret and Pig Models of Cystic Fibrosis: Prospects and Promise for Gene Therapy

(2015) Ziying Yan et al.   Human Gene Therapy Clinical Development

Improved vectors and genome-wide libraries for CRISPR screening

(2014) Neville E Sanjana et al.   NATURE METHODS

Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity

(2013) F. Ann Ran et al.   CELL

Postentry Processing of Recombinant Adeno-Associated Virus Type 1 and Transduction of the Ferret Lung Are Altered by a Factor in Airway Secretions

(2013) Ziying Yan et al.   HUMAN GENE THERAPY

A Novel Chimeric Adenoassociated Virus 2/Human Bocavirus 1 Parvovirus Vector Efficiently Transduces Human Airway Epithelia

(2013) Ziying Yan et al.   MOLECULAR THERAPY

Distinct transduction difference between adeno-associated virus type 1 and type 6 vectors in human polarized airway epithelia

(2012) Z Yan et al.   GENE THERAPY

Reduced airway surface pH impairs bacterial killing in the porcine cystic fibrosis lung

(2012) Alejandro A. Pezzulo et al.   NATURE

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

(2012) M. Jinek et al.   SCIENCE

A Versatile Viral System for Expression and Depletion of Proteins in Mammalian Cells

(2009) Eric Campeau et al.   PLoS One

Basal cells as stem cells of the mouse trachea and human airway epithelium

(2009) J. R. Rock et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

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

Mechanisms Governing Lentivirus Integration Site Selection

(2008) Angela Ciuffi   CURRENT GENE THERAPY