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List of works by R Scott McIvor

A Broad Range of Dose Optima Achieve High-level, Long-term Gene Expression After Hydrodynamic Delivery of Sleeping Beauty Transposons Using Hyperactive SB100x Transposase

scientific article

A facile method for somatic, lifelong manipulation of multiple genes in the mouse liver

scientific article

Adeno-associated virus type 2 vectors: transduction and long-term expression in cerebellar Purkinje cells in vivo is mediated by the fibroblast growth factor receptor 1 : bFGFR-1 mediates AAV2 transduction of Purkinje cells

scientific article

An ex vivo gene therapy approach to treat muscular dystrophy using inducible pluripotent stem cells ⬇️

scientific article published on January 1, 2013

Awakening gene therapy with Sleeping Beauty transposons

scientific article

Biodistribution of adeno-associated virus serotype 9 (AAV9) vector after intrathecal and intravenous delivery in mouse

scientific article

Cellular immune response against firefly luciferase after sleeping beauty-mediated gene transfer in vivo

scientific article

Characterization of the Human Artemis Promoter by Heterologous Gene Expression In Vitro and In Vivo ⬇️

scientific article published on June 10, 2011

Comparison of Endovascular and Intraventricular Gene Therapy With Adeno-Associated Virus-α-L-Iduronidase for Hurler Disease

scientific article

Conditional gene expression in the mouse using a Sleeping Beauty gene-trap transposon

scientific article

Correction of DNA protein kinase deficiency by spliceosome-mediated RNA trans-splicing and sleeping beauty transposon delivery

scientific article published on 24 April 2007

Counterselection and co-delivery of transposon and transposase functions for Sleeping Beauty-mediated transposition in cultured mammalian cells

scientific article published on December 2004

Cytotoxicity associated with artemis overexpression after lentiviral vector-mediated gene transfer

scientific article

Delivery of transposon DNA to lungs of mice using polyethyleneimine-DNA complexes

scientific article published on 01 January 2004

Differential adeno-associated virus mediated gene transfer to sensory neurons following intrathecal delivery by direct lumbar puncture

scientific article published on 28 May 2010

Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I ⬇️

scientific article published on March 17, 2011

Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing.

scientific article

Efficacy and Safety of Sleeping Beauty Transposon-Mediated Gene Transfer in Preclinical Animal Studies ⬇️

scientific article published on October 1, 2011

Efficient and stable transgene expression in human embryonic stem cells using transposon-mediated gene transfer

scientific article published on 2 August 2007

Efficient non-viral integration and stable gene expression in multipotent adult progenitor cells

scientific article published on 02 October 2011

Gene expression in lung and liver after intravenous infusion of polyethylenimine complexes of Sleeping Beauty transposons

scientific article

Gene insertion and long-term expression in lung mediated by the Sleeping Beauty transposon system

scientific article published in September 2003

Gene therapy for neurologic manifestations of mucopolysaccharidoses

scientific article

Harnessing a high cargo-capacity transposon for genetic applications in vertebrates

scientific article

Host factors that impact the biodistribution and persistence of multipotent adult progenitor cells

scientific article published on 12 January 2006

In vivo protection of activated Tyr22-dihydrofolate reductase gene-modified canine T lymphocytes from methotrexate.

scientific article published on June 2013

Inhibition of angiogenesis and suppression of colorectal cancer metastatic to the liver using the Sleeping Beauty Transposon System

scientific article

Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I.

scientific article published on 2 May 2017

Intravenous delivery for treatment of mucopolysaccharidosis type I: A comparison of AAV serotypes 9 and rh10

scientific article published on 20 May 2020

Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency

scientific article published on 9 September 2016

Liver-directed gene therapy using the sleeping beauty transposon system

scientific article

Long-term transgene expression and survival of transgene-expressing grafts following lentivirus transduction of bone marrow side population cells

scientific article published in April 2005

Lung-directed gene therapy in mice using the nonviral Sleeping Beauty transposon system

scientific article published in January 2007

Lysosomal enzyme can bypass the blood–brain barrier and reach the CNS following intranasal administration ⬇️

scientific article published on February 10, 2012

Methotrexate preconditioning allows sufficient engraftment to confer drug resistance in mice transplanted with marrow expressing drug-resistant dihydrofolate reductase activity

scientific article published on 27 April 2005

PR_b functionalized stealth liposomes for targeted delivery to metastatic colon cancer

scientific article published on 13 December 2012

Pax3-induced expansion enables the genetic correction of dystrophic satellite cells

scientific article

Preferential delivery of the Sleeping Beauty transposon system to livers of mice by hydrodynamic injection

scientific article

Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System-Directed, AAV9-Mediated Iduronate Sulfatase Gene Transfer

scientific article published on 6 May 2017

Prolonged Expression of Secreted Enzymes in Dogs After Liver-Directed Delivery of Sleeping Beauty Transposons: Implications for Non-Viral Gene Therapy of Systemic Disease

scientific article published on 20 May 2017

Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses

scientific article

Protection of mice from methotrexate toxicity by ex vivo transduction using lentivirus vectors expressing drug-resistant dihydrofolate reductase

scientific article published on 22 June 2007

Quantitative analysis of α-L-iduronidase expression in immunocompetent mice treated with the Sleeping Beauty transposon system

scientific article

Role of transgene regulation in ex vivo lentiviral correction of artemis deficiency

scientific article published on 13 April 2015

Simple and efficient methods for enrichment and isolation of endonuclease modified cells

scientific article (publication date: 2014)

Sleeping Beauty transposon-mediated engineering of human primary T cells for therapy of CD19+ lymphoid malignancies

scientific article

Sleeping Beauty-Mediated Drug Resistance Gene Transfer in Human Hematopoietic Progenitor Cells

scientific article

Sleeping Beauty-mediated correction of Fanconi anemia type C.

scientific article

Sleeping Beauty-mediated transposition and long-term expression in vivo: use of the LoxP/Cre recombinase system to distinguish transposition-specific expression

scientific article published on 13 December 2005

Sleeping beauty transposition from nonintegrating lentivirus

scientific article

Sleeping beauty transposon-mediated gene therapy for prolonged expression

scientific article

Somatic integration of an oncogene-harboring Sleeping Beauty transposon models liver tumor development in the mouse

scientific article

Supraspinal gene transfer by intrathecal adeno-associated virus serotype 5.

scientific article

Systemic correction of storage disease in MPS I NOD/SCID mice using the sleeping beauty transposon system

scientific article

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

scientific article published on April 15, 2011

ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

scientific article published on 01 November 2018

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