Tailored Pig Models for Preclinical Efficacy and Safety Testing of Targeted Therapies (Q38618881)

4 September 2017

Tailored Pig Models for Preclinical Efficacy and Safety Testing of Targeted Therapies (English)

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4 September 2017

6

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4 September 2017

Nikolai Klymiuk

1

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4 September 2017

Frank Seeliger

2

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4 September 2017

Mohammad Bohlooly-Y

3

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4 September 2017

Andreas Blutke

4

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4 September 2017

Daniel G Rudmann

5

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4 September 2017

language of work or name

English

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publication date

27 October 2015

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4 September 2017

Toxicologic Pathology

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4 September 2017

44

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4 September 2017

3

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4 September 2017

346-357

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Transgenic pigs as models for translational biomedical research

4 September 2017

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https://api.crossref.org/works/10.1177%2F0192623315609688

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Targeted disruption of exon 52 in the mouse dystrophin gene induced muscle degeneration similar to that observed in Duchenne muscular dystrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

The AAV vector toolkit: poised at the clinical crossroads

21 January 2018

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https://api.crossref.org/works/10.1177%2F0192623315609688

Rare-disease genetics in the era of next-generation sequencing: discovery to translation

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

The mammalian gene function resource: the International Knockout Mouse Consortium

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Human monogenic disorders - a source of novel drug targets

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Ataluren treatment of patients with nonsense mutation dystrophinopathy

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Efficient gene delivery to pig airway epithelia and submucosal glands using helper-dependent adenoviral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Efficient TALEN-mediated gene knockout in livestock

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Sinus hypoplasia precedes sinus infection in a porcine model of cystic fibrosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Loss of anion transport without increased sodium absorption characterizes newborn porcine cystic fibrosis airway epithelia

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Functional disruption of the dystrophin gene in rhesus monkey using CRISPR/Cas9.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

The CFTR and ENaC debate: how important is ENaC in CF lung disease?

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Genome editing technologies: defining a path to clinic

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Cystic fibrosis genetics: from molecular understanding to clinical application

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Targeted disruption of the alpha1,3-galactosyltransferase gene in cloned pigs

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

The era of genomic medicine.

21 January 2018

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https://api.crossref.org/works/10.1177%2F0192623315609688

CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Sonographic evidence of abnormal tracheal cartilage ring structure in cystic fibrosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Pig models of neurodegenerative disorders: Utilization in cell replacement-based preclinical safety and efficacy studies.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Inflammation in cystic fibrosis--when and why? Friend or foe?

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Financing drug discovery for orphan diseases

21 January 2018

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https://api.crossref.org/works/10.1177%2F0192623315609688

Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Read-through strategies for suppression of nonsense mutations in Duchenne/ Becker muscular dystrophy: aminoglycosides and ataluren (PTC124).

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

The new pig on the block: modelling cancer in pigs

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Cystic fibrosis gene therapy: successes, failures and hopes for the future

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

A novel gene delivery method transduces porcine pancreatic duct epithelial cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Analyses of pig genomes provide insight into porcine demography and evolution

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Efficient intratracheal delivery of airway epithelial cells in mice and pigs

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

One-step generation of knockout pigs by zygote injection of CRISPR/Cas system

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Adeno-associated virus (AAV) vectors in gene therapy: immune challenges and strategies to circumvent them.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Efficient generation of a biallelic knockout in pigs using zinc-finger nucleases

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Impaired mucus detachment disrupts mucociliary transport in a piglet model of cystic fibrosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Dystrophin: the protein product of the Duchenne muscular dystrophy locus

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Development and applications of CRISPR-Cas9 for genome engineering

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

INFRAFRONTIER--providing mutant mouse resources as research tools for the international scientific community

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Human cystic fibrosis airway epithelia have reduced Cl- conductance but not increased Na+ conductance.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Targeting efficiency of a-1,3-galactosyl transferase gene in pig fetal fibroblast cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Gastrointestinal Manifestations of Cystic Fibrosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

A guide to genome engineering with programmable nucleases

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Dystrophin-deficient pigs provide new insights into the hierarchy of physiological derangements of dystrophic muscle

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

First inducible transgene expression in porcine large animal models

21 January 2018

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https://api.crossref.org/works/10.1177%2F0192623315609688

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Production of alpha-1,3-galactosyltransferase knockout pigs by nuclear transfer cloning.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Characterization of dystrophin deficient rats: a new model for Duchenne muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Loss of cystic fibrosis transmembrane conductance regulator function produces abnormalities in tracheal development in neonatal pigs and young children

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Mammalian models of Duchenne Muscular Dystrophy: pathological characteristics and therapeutic applications

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Generation of muscular dystrophy model rats with a CRISPR/Cas system

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Genetic medicines: treatment strategies for hereditary disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

The ΔF508 mutation causes CFTR misprocessing and cystic fibrosis-like disease in pigs

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

The mdx mouse model as a surrogate for Duchenne muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Production of CFTR-null and CFTR-DeltaF508 heterozygous pigs by adeno-associated virus-mediated gene targeting and somatic cell nuclear transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Disruption of the CFTR gene produces a model of cystic fibrosis in newborn pigs

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

CRISPR-Cas systems for editing, regulating and targeting genomes

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

CRISPR genome engineering and viral gene delivery: a case of mutual attraction

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Functional repair of CFTR by CRISPR/Cas9 in intestinal stem cell organoids of cystic fibrosis patients

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Gene therapy finds its niche

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Cardiomyopathy of Duchenne muscular dystrophy: current understanding and future directions

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Cystic fibrosis pigs develop lung disease and exhibit defective bacterial eradication at birth

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Intestinal CFTR expression alleviates meconium ileus in cystic fibrosis pigs

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Progress and problems with the use of viral vectors for gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Characterization of defects in ion transport and tissue development in cystic fibrosis transmembrane conductance regulator (CFTR)-knockout rats

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Mouse models of cystic fibrosis: phenotypic analysis and research applications

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Genetically engineered pig models for diabetes research

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Ferret and pig models of cystic fibrosis: prospects and promise for gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

Non-viral vectors for gene-based therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F0192623315609688

21 January 2018

Identifiers

DOI

10.1177/0192623315609688

1 reference

4 September 2017

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