Recent developments in transposon-mediated gene therapy

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

Genetically Modified T cells Targeting Interleukin-11 Receptor  -Chain Kill Human Osteosarcoma Cells and Induce the Regression of Established Osteosarcoma Lung Metastases

(2011) G. Huang et al.   CANCER RESEARCH

Immune Responses to AAV in Clinical Trials

(2011) Federico Mingozzi et al.   CURRENT GENE THERAPY

The hyperactive Sleeping Beauty transposase SB100X improves the genetic modification of T cells to express a chimeric antigen receptor

(2011) Z Jin et al.   GENE THERAPY

Nonviral Gene Delivery with the Sleeping Beauty Transposon System

(2011) Zoltán Ivics et al.   HUMAN GENE THERAPY

Hyperactive piggyBac Gene Transfer in Human Cells and In Vivo

(2011) Joseph E. Doherty et al.   HUMAN GENE THERAPY

The Sleeping Beauty transposon system: a non-viral vector for gene therapy

(2011) E. L. Aronovich et al.   HUMAN MOLECULAR GENETICS

Manipulating piggyBac Transposon Chromosomal Integration Site Selection in Human Cells

(2011) Claudia Kettlun et al.   MOLECULAR THERAPY

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

(2011) Yozo Nakazawa et al.   MOLECULAR THERAPY

Comparative Genomic Integration Profiling of Sleeping Beauty Transposons Mobilized With High Efficacy From Integrase-defective Lentiviral Vectors in Primary Human Cells

(2011) Brian Moldt et al.   MOLECULAR THERAPY

Combining mTor Inhibitors With Rapamycin-resistant T Cells: A Two-pronged Approach to Tumor Elimination

(2011) Leslie E Huye et al.   MOLECULAR THERAPY

Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells

(2011) Kosuke Yusa et al.   NATURE

A more efficient method to generate integration-free human iPS cells

(2011) Keisuke Okita et al.   NATURE METHODS

Mobilization of giant piggyBac transposons in the mouse genome

(2011) Meng Amy Li et al.   NUCLEIC ACIDS RESEARCH

Avoiding cytotoxicity of transposases by dose-controlled mRNA delivery

(2011) Melanie Galla et al.   NUCLEIC ACIDS RESEARCH

Erythroid-Specific Expression of β-globin from Sleeping Beauty-Transduced Human Hematopoietic Progenitor Cells

(2011) Lucas M. Sjeklocha et al.   PLoS One

Efficient generation of transgene-free human induced pluripotent stem cells (iPSCs) by temperature-sensitive Sendai virus vectors

(2011) H. Ban et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The expanding universe of transposon technologies for gene and cell engineering

(2011) Zoltán Ivics et al.   Mobile DNA

Translating Sleeping Beauty transposition into cellular therapies: Victories and challenges

(2010) Zsuzsanna Izsvák et al.   BIOESSAYS

Highly Efficient Reprogramming to Pluripotency and Directed Differentiation of Human Cells with Synthetic Modified mRNA

(2010) Luigi Warren et al.   Cell Stem Cell

Herpes Simplex Virus/Sleeping Beauty Vector-Based Embryonic Gene Transfer Using the HSB5 Mutant: Loss of Apparent Transposition Hyperactivity In Vivo

(2010) Suresh de Silva et al.   HUMAN GENE THERAPY

Virion-Associated Cofactor High-Mobility Group DNA-Binding Protein-1 Facilitates Transposition from the Herpes Simplex Virus/Sleeping Beauty Amplicon Vector Platform

(2010) Suresh de Silva et al.   HUMAN GENE THERAPY

piggyBac Transposon-mediated Long-term Gene Expression in Mice

(2010) Hideyuki Nakanishi et al.   MOLECULAR THERAPY

Duration of Expression and Activity of Sleeping Beauty Transposase in mouse liver following hydrodynamic DNA delivery

(2010) Jason B Bell et al.   MOLECULAR THERAPY

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

Comparative Analysis of Transposable Element Vector Systems in Human Cells

(2010) Ivana Grabundzija et al.   MOLECULAR THERAPY

Gene Transfer Efficiency and Genome-Wide Integration Profiling of Sleeping Beauty, Tol2, and PiggyBac Transposons in Human Primary T Cells

(2010) Xin Huang et al.   MOLECULAR THERAPY

A Transposon and Transposase System for Human Application

(2010) Perry B Hackett et al.   MOLECULAR THERAPY

Generation of adult human induced pluripotent stem cells using nonviral minicircle DNA vectors

(2010) Kazim H Narsinh et al.   Nature Protocols

Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency

(2010) Salima Hacein-Bey-Abina et al.   NEW ENGLAND JOURNAL OF MEDICINE

Impact of Hydrodynamic Injection and phiC31 Integrase on Tumor Latency in a Mouse Model of MYC-Induced Hepatocellular Carcinoma

(2010) Lauren E. Woodard et al.   PLoS One

Novel Hyperactive Transposons for Genetic Modification of Induced Pluripotent and Adult Stem Cells: A Nonviral Paradigm for Coaxed Differentiation

(2010) Eyayu Belay et al.   STEM CELLS

Emerging potential of transposons for gene therapy and generation of induced pluripotent stem cells

(2009) T. VandenDriessche et al.   BLOOD

Stable gene transfer and expression in cord blood-derived CD34+ hematopoietic stem and progenitor cells by a hyperactive Sleeping Beauty transposon system

(2009) X. Xue et al.   BLOOD

Transposon-mediated BAC transgenesis in zebrafish and mice

(2009) Maximiliano L Suster et al.   BMC GENOMICS

An Efficient and Reversible Transposable System for Gene Delivery and Lineage-Specific Differentiation in Human Embryonic Stem Cells

(2009) Arnaud Lacoste et al.   Cell Stem Cell

Generation of Human Induced Pluripotent Stem Cells by Direct Delivery of Reprogramming Proteins

(2009) Dohoon Kim et al.   Cell Stem Cell

Indoleamine 2,3-dioxygenase attenuates inhibitor development in gene-therapy-treated hemophilia A mice

(2009) L Liu et al.   GENE THERAPY

Chromosomal mobilization and reintegration ofSleeping BeautyandPiggyBactransposons

(2009) Qi Liang et al.   GENESIS

Efficient Transposition of Tol2 in the Mouse Germline

(2009) Vincent W. Keng et al.   GENETICS

Long-term reduction of jaundice in Gunn rats by nonviral liver-targeted delivery of Sleeping Beauty transposon

(2009) Xia Wang et al.   HEPATOLOGY

Gene Therapy Delivers

(2009) Thierry VandenDriessche   HUMAN GENE THERAPY

Stable Transgene Expression in Primitive Human CD34+ Hematopoietic Stem/Progenitor Cells, Using the Sleeping Beauty Transposon System

(2009) Teiko Sumiyoshi et al.   HUMAN GENE THERAPY

piggyBac Transposon/Transposase System to Generate CD19-Specific T Cells for the Treatment of B-Lineage Malignancies

(2009) Pallavi V. Raja Manuri et al.   HUMAN GENE THERAPY

Gene Expression in Lung and Liver After Intravenous Infusion of Polyethylenimine Complexes of Sleeping Beauty Transposons

(2009) Kelly M. Podetz-Pedersen et al.   HUMAN GENE THERAPY

Cardiac cell therapy: overexpression of connexin43 in skeletal myoblasts and prevention of ventricular arrhythmias

(2009) Sarah Fernandes et al.   JOURNAL OF CELLULAR AND MOLECULAR MEDICINE

Preventing and exploiting the oncogenic potential of integrating gene vectors

(2009) Ute Modlich et al.   JOURNAL OF CLINICAL INVESTIGATION

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

(2009) Eugenio Montini et al.   JOURNAL OF CLINICAL INVESTIGATION

Genome-wide Mapping of PiggyBac Transposon Integrations in Primary Human T Cells

(2009) Daniel L. Galvan et al.   JOURNAL OF IMMUNOTHERAPY

PiggyBac Transposon-based Inducible Gene Expression In Vivo After Somatic Cell Gene Transfer

(2009) Sai K Saridey et al.   MOLECULAR THERAPY

Hybrid Lentivirus-transposon Vectors With a Random Integration Profile in Human Cells

(2009) Nicklas H Staunstrup et al.   MOLECULAR THERAPY

Sleeping Beauty Transposition From Nonintegrating Lentivirus

(2009) Conrad A Vink et al.   MOLECULAR THERAPY

Systemic Correction of Storage Disease in MPS I NOD/SCID Mice Using the Sleeping Beauty Transposon System

(2009) Elena L Aronovich et al.   MOLECULAR THERAPY

Virus-free induction of pluripotency and subsequent excision of reprogramming factors

(2009) Keisuke Kaji et al.   NATURE

piggyBac transposition reprograms fibroblasts to induced pluripotent stem cells

(2009) Knut Woltjen et al.   NATURE

Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates

(2009) Lajos Mátés et al.   NATURE GENETICS

Generation of transgene-free induced pluripotent mouse stem cells by the piggyBac transposon

(2009) Kosuke Yusa et al.   NATURE METHODS

An adenovirus-delivered peptide aptamer C1-1 targeting the core protein of hepatitis B virus inhibits viral DNA replication and production in vitro and in vivo

(2009) Wei Zhang et al.   PEPTIDES

Directed Differentiation of Human-Induced Pluripotent Stem Cells Generates Active Motor Neurons

(2009) Saravanan Karumbayaram et al.   STEM CELLS

Are transposable elements simply silenced or are they under house arrest?

(2009) Christian Biémont   TRENDS IN GENETICS

Ten years of gene therapy for primary immune deficiencies

(2009) A. Aiuti et al.   Hematology-American Society of Hematology Education Program

Resistance of mature T cells to oncogene transformation

(2008) S. Newrzela et al.   BLOOD

Minimally invasive and selective hydrodynamic gene therapy of liver segments in the pig and human

(2008) S E Khorsandi et al.   CANCER GENE THERAPY

Redirecting Specificity of T-Cell Populations For CD19 Using the Sleeping Beauty System

(2008) H. Singh et al.   CANCER RESEARCH

piggyBac can bypass DNA synthesis during cut and paste transposition

(2008) Rupak Mitra et al.   EMBO JOURNAL

Success in sight: The eyes have it! Ocular gene therapy trials for LCA look promising

(2008) J W Bainbridge et al.   GENE THERAPY

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION

Targeted gene insertion for molecular medicine

(2008) Katrin Voigt et al.   JOURNAL OF MOLECULAR MEDICINE-JMM

Sleeping Beauty Vector System Moves Toward Human Trials in the United States

(2008) David A Williams   MOLECULAR THERAPY

Computer-assisted Hydrodynamic Gene Delivery

(2008) Takeshi Suda et al.   MOLECULAR THERAPY

Sleeping Beauty Transposon‐mediated Engineering of Human Primary T Cells for Therapy of CD19+ Lymphoid Malignancies

(2008) Xin Huang et al.   MOLECULAR THERAPY

Shielding of Sleeping Beauty DNA Transposon-delivered Transgene Cassettes by Heterologous Insulators in Early Embryonal Cells

(2008) Trine Dalsgaard et al.   MOLECULAR THERAPY

Induced Pluripotent Stem Cells Generated Without Viral Integration

(2008) M. Stadtfeld et al.   SCIENCE

Generation of Mouse Induced Pluripotent Stem Cells Without Viral Vectors

(2008) K. Okita et al.   SCIENCE

The Therapeutic Potential of Embryonic and Adult Stem Cells for Skeletal Muscle Regeneration

(2008) Radbod Darabi et al.   Stem Cell Reviews and Reports

Stable gene transfer of CCR5 and CXCR4 siRNAs by sleeping beauty transposon system to confer HIV-1 resistance

(2008) Mayur Tamhane et al.   AIDS Research and Therapy

DNA-binding Specificity Is a Major Determinant of the Activity and Toxicity of Zinc-finger Nucleases

(2007) Tatjana I Cornu et al.   MOLECULAR THERAPY

Transcriptional Activities of the Sleeping Beauty Transposon and Shielding Its Genetic Cargo With Insulators

(2007) Oliver Walisko et al.   MOLECULAR THERAPY