Targeted Integration of a Super-Exon into the CFTR Locus Leads to Functional Correction of a Cystic Fibrosis Cell Line Model

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

(2016) Ciaran M Lee et al.   MOLECULAR THERAPY

Streptococcus thermophilus CRISPR-Cas9 Systems Enable Specific Editing of the Human Genome

(2016) Maximilian Müller et al.   MOLECULAR THERAPY

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

(2016) Benjamin P. Kleinstiver et al.   NATURE

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

TALEN-mediated functional correction of X-linked chronic granulomatous disease in patient-derived induced pluripotent stem cells

(2015) Anne-Kathrin Dreyer et al.   BIOMATERIALS

Differential contribution ofcis-regulatory elements to higher order chromatin structure and expression of theCFTRlocus

(2015) Rui Yang et al.   NUCLEIC ACIDS RESEARCH

Rationally engineered Cas9 nucleases with improved specificity

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

Protein Traffic Disorders: an Effective High-Throughput Fluorescence Microscopy Pipeline for Drug Discovery

(2015) Hugo M. Botelho et al.   Scientific Reports

Chromatin Dynamics in the Regulation of CFTR Expression

(2015) Nehal Gosalia et al.   Genes

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

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

(2014) Pablo Tebas et al.   NEW ENGLAND JOURNAL OF MEDICINE

TALENs facilitate targeted genome editing in human cells with high specificity and low cytotoxicity

(2014) Claudio Mussolino et al.   NUCLEIC ACIDS RESEARCH

One-Step Generation of Mice Carrying Mutations in Multiple Genes by CRISPR/Cas-Mediated Genome Engineering

(2013) Haoyi Wang et al.   CELL

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

(2013) Gerald Schwank et al.   Cell Stem Cell

Rescuing Mutant CFTR: A Multi-task Approach to a Better Outcome in Treating Cystic Fibrosis

(2013) Margarida D. Amaral et al.   CURRENT PHARMACEUTICAL DESIGN

Efficient Clinical Scale Gene Modification via Zinc Finger Nuclease–Targeted Disruption of the HIV Co-receptor CCR5

(2013) Dawn A. Maier et al.   HUMAN GENE THERAPY

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

(2013) Patrick R Sosnay et al.   NATURE GENETICS

A functional CFTR assay using primary cystic fibrosis intestinal organoids

(2013) Johanna F Dekkers et al.   NATURE MEDICINE

Nucleosome mapping across the CFTR locus identifies novel regulatory factors

(2013) Erbay Yigit et al.   NUCLEIC ACIDS RESEARCH

The Genomic and Transcriptomic Landscape of a HeLa Cell Line

(2013) Jonathan J. M. Landry et al.   G3-Genes Genomes Genetics

The Nontoxic Cell Cycle Modulator Indirubin Augments Transduction of Adeno-Associated Viral Vectors and Zinc-Finger Nuclease-Mediated Gene Targeting

(2012) Shamim H. Rahman et al.   HUMAN GENE THERAPY

Generation and Genetic Engineering of Human Induced Pluripotent Stem Cells Using Designed Zinc Finger Nucleases

(2012) Sivaprakash Ramalingam et al.   STEM CELLS AND DEVELOPMENT

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

(2011) Hojun Li et al.   NATURE

Genetic engineering of human pluripotent cells using TALE nucleases

(2011) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

Revealing off-target cleavage specificities of zinc-finger nucleases by in vitro selection

(2011) Vikram Pattanayak et al.   NATURE METHODS

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

(2011) Claudio Mussolino et al.   NUCLEIC ACIDS RESEARCH

Collaboration and competition between DNA double-strand break repair pathways

(2010) Elizabeth M. Kass et al.   FEBS LETTERS

Correction of the  Phe508 Cystic Fibrosis Transmembrane Conductance Regulator Trafficking Defect by the Bioavailable Compound Glafenine

(2010) R. Robert et al.   MOLECULAR PHARMACOLOGY

Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo

(2010) Nathalia Holt et al.   NATURE BIOTECHNOLOGY

Autonomous zinc-finger nuclease pairs for targeted chromosomal deletion

(2010) Cem Şöllü et al.   NUCLEIC ACIDS RESEARCH

A truncated CFTR protein rescues endogenous ΔF508-CFTR and corrects chloride transport in mice

(2009) Estelle Cormet-Boyaka et al.   FASEB JOURNAL

Fate of Recombinant Adeno-Associated Viral Vector Genomes During DNA Double-Strand Break-Induced Gene Targeting in Human Cells

(2009) Katharina Gellhaus et al.   HUMAN GENE THERAPY

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

Activation of the Unfolded Protein Response by ΔF508 CFTR

(2008) Rafal Bartoszewski et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Rapid “Open-Source” Engineering of Customized Zinc-Finger Nucleases for Highly Efficient Gene Modification

(2008) Morgan L. Maeder et al.   MOLECULAR CELL

Impact of non-homologous end-joining deficiency on random and targeted DNA integration: implications for gene targeting

(2008) Susumu Iiizumi et al.   NUCLEIC ACIDS RESEARCH