Modeling correction of severe urea cycle defects in the growing murine liver using a hybrid recombinant adeno-associated virus/piggyBactransposase gene delivery system

Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy

(2015) Randy J. Chandler et al.   JOURNAL OF CLINICAL INVESTIGATION

Hybrid Nonviral/Viral Vector Systems for Improved piggyBac DNA Transposon In Vivo Delivery

(2015) Ashley L Cooney et al.   MOLECULAR THERAPY

A longitudinal study of urea cycle disorders

(2014) Mark L. Batshaw et al.   MOLECULAR GENETICS AND METABOLISM

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Chromatin Landscapes of Retroviral and Transposon Integration Profiles

(2014) Johann de Jong et al.   PLoS Genetics

Mechanisms of Retroviral Integration and Mutagenesis

(2013) Alessia Cavazza et al.   HUMAN GENE THERAPY

Adeno-associated Virus-mediated Rescue of Neonatal Lethality in Argininosuccinate Synthetase-deficient Mice

(2013) Cindy Y Kok et al.   MOLECULAR THERAPY

Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

(2013) Leszek Lisowski et al.   NATURE

Hybrid Adeno-Associated Viral Vectors Utilizing Transposase-Mediated Somatic Integration for Stable Transgene Expression in Human Cells

(2013) Wenli Zhang et al.   PLoS One

Comparison of gene transfer to the murine liver following intraperitoneal and intraportal delivery of hepatotropic AAV pseudo-serotypes

(2012) A P Dane et al.   GENE THERAPY

Suggested guidelines for the diagnosis and management of urea cycle disorders

(2012) Johannes Häberle et al.   Orphanet Journal of Rare Diseases

Induction of hepatocellular carcinoma by in vivo gene targeting

(2012) P.-R. Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Genome-wide target profiling of piggyBac and Tol2 in HEK 293: pros and cons for gene discovery and gene therapy

(2011) Yaa-Jyuhn J Meir et al.   BMC BIOTECHNOLOGY

Distribution of Lentiviral Vector Integration Sites in Mice Following Therapeutic Gene Transfer to Treat β-thalassemia

(2011) Keshet Ronen et al.   MOLECULAR THERAPY

Induction and Prevention of Severe Hyperammonemia in the spfash Mouse Model of Ornithine Transcarbamylase Deficiency Using shRNA and rAAV-mediated Gene Delivery

(2011) Sharon C Cunningham et al.   MOLECULAR THERAPY

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

BEDTools: a flexible suite of utilities for comparing genomic features

(2010) Aaron R. Quinlan et al.   BIOINFORMATICS

High-definition mapping of retroviral integration sites identifies active regulatory elements in human multipotent hematopoietic progenitors

(2010) C. Cattoglio et al.   BLOOD

Insertional Mutagenesis in Mice Deficient for p15Ink4b, p16Ink4a, p21Cip1, and p27Kip1 Reveals Cancer Gene Interactions and Correlations with Tumor Phenotypes

(2010) J. Kool et al.   CANCER RESEARCH

Hyperactive Sleeping Beauty Transposase Enables Persistent Phenotypic Correction in Mice and a Canine Model for Hemophilia B

(2010) Martin A Hausl et al.   MOLECULAR THERAPY

Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

(2010) Stefan Stein et al.   NATURE MEDICINE

Identification of Potential Driver Genes in Human Liver Carcinoma by Genomewide Screening

(2009) H. G. Woo et al.   CANCER RESEARCH

AAV2/8-mediated Correction of OTC Deficiency Is Robust in Adult but Not Neonatal Spfash Mice

(2009) Sharon C Cunningham et al.   MOLECULAR THERAPY

Ultrafast and memory-efficient alignment of short DNA sequences to the human genome

(2009) Ben Langmead et al.   GENOME BIOLOGY

Large-Scale Mutagenesis in p19ARF- and p53-Deficient Mice Identifies Cancer Genes and Their Collaborative Networks

(2008) Anthony G. Uren et al.   CELL

Contrasting features of urea cycle disorders in human patients and knockout mouse models

(2008) Joshua L. Deignan et al.   MOLECULAR GENETICS AND METABOLISM

Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors

(2008) Sharon C Cunningham et al.   MOLECULAR THERAPY

Chromosomal transposition of PiggyBac in mouse embryonic stem cells

(2008) W. Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA