Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus

Towards next generation therapies for cystic fibrosis: Folding, function and pharmacology of CFTR

(2020) Samuel J. Bose et al.   Journal of Cystic Fibrosis

AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9

(2020) Ang Li et al.   MOLECULAR 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

Challenges Facing Airway Epithelial Cell-Based Therapy for Cystic Fibrosis

(2019) Andrew Berical et al.   Frontiers in Pharmacology

A New Class of Medicines through DNA Editing

(2019) Matthew H. Porteus   NEW ENGLAND JOURNAL OF MEDICINE

Identification of preexisting adaptive immunity to Cas9 proteins in humans

(2019) Carsten T. Charlesworth et al.   NATURE MEDICINE

Highly Efficient and Marker-free Genome Editing of Human Pluripotent Stem Cells by CRISPR-Cas9 RNP and AAV6 Donor-Mediated Homologous Recombination

(2019) Renata M. Martin et al.   Cell Stem Cell

Gene correction for SCID-X1 in long-term hematopoietic stem cells

(2019) Mara Pavel-Dinu et al.   Nature Communications

Clinical care for cystic fibrosis: preparing for the future now

(2019) Michael W Konstan et al.   Lancet Respiratory Medicine

Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial

(2019) Harry G M Heijerman et al.   LANCET

Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I

(2019) Natalia Gomez-Ospina et al.   Nature Communications

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

An Activity Switch in Human Telomerase Based on RNA Conformation and Shaped by TCAB1

(2018) Lu Chen et al.   CELL

Distributed hepatocytes expressing telomerase repopulate the liver in homeostasis and injury

(2018) Shengda Lin et al.   NATURE

p53 inhibits CRISPR–Cas9 engineering in human pluripotent stem cells

(2018) Robert J. Ihry et al.   NATURE MEDICINE

CRISPR–Cas9 genome editing induces a p53-mediated DNA damage response

(2018) Emma Haapaniemi et al.   NATURE MEDICINE

CRISPR/Cas9 genome editing in human hematopoietic stem cells

(2018) Rasmus O Bak et al.   Nature Protocols

Priming Human Repopulating Hematopoietic Stem and Progenitor Cells for Cas9/sgRNA Gene Targeting

(2018) Carsten T. Charlesworth et al.   Molecular Therapy-Nucleic Acids

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

(2018) Christopher A. Vakulskas et al.   NATURE MEDICINE

High prevalence of Streptococcus pyogenes Cas9-reactive T cells within the adult human population

(2018) Dimitrios L. Wagner et al.   NATURE MEDICINE

VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles

(2018) Dominic Keating et al.   NEW ENGLAND JOURNAL OF MEDICINE

Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del

(2017) Jennifer L. Taylor-Cousar et al.   NEW ENGLAND JOURNAL OF MEDICINE

Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy

(2017) Leonela Amoasii et al.   Science Translational Medicine

CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor Vectors

(2017) Rasmus O. Bak et al.   Cell Reports

Genome Editing: A New Approach to Human Therapeutics

(2016) Matthew Porteus   Annual Review of Pharmacology and Toxicology

Dual SMAD Signaling Inhibition Enables Long-Term Expansion of Diverse Epithelial Basal Cells

(2016) Hongmei Mou et al.   Cell Stem Cell

The Neisseria meningitidis CRISPR-Cas9 System Enables Specific Genome Editing in Mammalian Cells

(2016) Ciaran M Lee et al.   MOLECULAR THERAPY

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells

(2016) Daniel P. Dever et al.   NATURE

Relationships among CFTR expression, HCO3−secretion, and host defense may inform gene- and cell-based cystic fibrosis therapies

(2016) Viral S. Shah et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

Sequence artefacts in a prospective series of formalin-fixed tumours tested for mutations in hotspot regions by massively parallel sequencing

(2014) Stephen Q Wong et al.   BMC Medical Genomics

An ultrasensitive method for quantitating circulating tumor DNA with broad patient coverage

(2014) Aaron M Newman et al.   NATURE MEDICINE

A Little CFTR Goes a Long Way: CFTR-Dependent Sweat Secretion from G551D and R117H-5T Cystic Fibrosis Subjects Taking Ivacaftor

(2014) Jessica E. Char et al.   PLoS One

Generation of multiciliated cells in functional airway epithelia from human induced pluripotent stem cells

(2014) A. L. Firth et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

COSMID: A Web-based Tool for Identifying and Validating CRISPR/Cas Off-target Sites

(2014) Thomas J Cradick et al.   Molecular Therapy-Nucleic Acids

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

(2013) Gerald Schwank et al.   Cell Stem Cell

Targeted, High-Depth, Next-Generation Sequencing of Cancer Genes in Formalin-Fixed, Paraffin-Embedded and Fine-Needle Aspiration Tumor Specimens

(2013) Andrew G. Hadd et al.   JOURNAL OF MOLECULAR DIAGNOSTICS

CD4+ T Cells from IPEX Patients Convert into Functional and Stable Regulatory T Cells by FOXP3 Gene Transfer

(2013) L. Passerini et al.   Science Translational Medicine

Engineered human Tmpk fused with truncated cell-surface markers: versatile cell-fate control safety cassettes

(2012) M Scaife et al.   GENE THERAPY

PiggyBac-mediated Cancer Immunotherapy Using EBV-specific Cytotoxic T-cells Expressing HER2-specific Chimeric Antigen Receptor

(2011) Yozo Nakazawa et al.   MOLECULAR THERAPY

Site-specific integration and tailoring of cassette design for sustainable gene transfer

(2011) Angelo Lombardo et al.   NATURE METHODS

Telomere length measurement by a novel monochrome multiplex quantitative PCR method

(2009) R. M. Cawthon   NUCLEIC ACIDS RESEARCH

Intronic enhancers coordinate epithelial-specific looping of the active CFTR locus

(2009) C. J. Ott 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

Functional Properties of Mixed Cystic Fibrosis and Normal Bronchial Epithelial Cell Cultures

(2008) Luc Dannhoffer et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY