Literature DB >> 21459777

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

Elena L Aronovich1, R Scott McIvor, Perry B Hackett.   

Abstract

Over the past decade, the Sleeping Beauty (SB) transposon system has been developed as the leading non-viral vector for gene therapy. This vector combines the advantages of viruses and naked DNA. Here we review progress over the last 2 years in vector design, methods of delivery and safety that have supported its use in the clinic. Currently, the SB vector has been validated for ex vivo gene delivery to stem cells, including T-cells for the treatment of lymphoma. Progress in delivery of SB transposons to liver for treatment of various systemic diseases, such as hemophilia and mucopolysaccharidoses types I and VII, has encountered some problems, but even here progress is being made.

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Year:  2011        PMID： 21459777      PMCID： PMC3095056          DOI： 10.1093/hmg/ddr140

Source DB:  PubMed          Journal:  Hum Mol Genet        ISSN： 0964-6906            Impact factor:   6.150


  89 in total

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

Authors:  Andrew Wilber; Jonathan L Linehan; Xinghui Tian; Petter S Woll; Julie K Morris; Lalitha R Belur; R Scott McIvor; Dan S Kaufman
Journal:  Stem Cells       Date:  2007-08-02       Impact factor: 6.277

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

Authors:  Elena L Aronovich; Jason B Bell; Lalitha R Belur; Roland Gunther; Brenda Koniar; David C C Erickson; Patricia A Schachern; Ilze Matise; R Scott McIvor; Chester B Whitley; Perry B Hackett
Journal:  J Gene Med       Date:  2007-05       Impact factor: 4.565

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

Authors:  Jason B Bell; Kelly M Podetz-Pedersen; Elena L Aronovich; Lalitha R Belur; R Scott McIvor; Perry B Hackett
Journal:  Nat Protoc       Date:  2007       Impact factor: 13.491

4.  Hydrodynamic gene delivery to the pig liver via an isolated segment of the inferior vena cava.

Authors:  J W Fabre; A Grehan; M Whitehorne; G J Sawyer; X Dong; S Salehi; L Eckley; X Zhang; M Seddon; A M Shah; M Davenport; M Rela
Journal:  Gene Ther       Date:  2007-11-15       Impact factor: 5.250

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

Authors:  Xin Huang; Hongfeng Guo; Johnthomas Kang; Suet Choi; Tom C Zhou; Syam Tammana; Christopher J Lees; Zhong-Ze Li; Michael Milone; Bruce L Levine; Jakub Tolar; Carl H June; R Scott McIvor; John E Wagner; Bruce R Blazar; Xianzheng Zhou
Journal:  Mol Ther       Date:  2008-01-29       Impact factor: 11.454

6.  Messenger RNA as a source of transposase for sleeping beauty transposon-mediated correction of hereditary tyrosinemia type I.

Authors:  Andrew Wilber; Kirk J Wangensteen; Yixin Chen; Lijuan Zhuo; Joel L Frandsen; Jason B Bell; Zongyu J Chen; Stephen C Ekker; R Scott McIvor; Xin Wang
Journal:  Mol Ther       Date:  2007-04-17       Impact factor: 11.454

7.  Targeted Sleeping Beauty transposition in human cells.

Authors:  Zoltán Ivics; Andrea Katzer; Eva E Stüwe; Dora Fiedler; Siegne Knespel; Zsuzsanna Izsvák
Journal:  Mol Ther       Date:  2007-04-10       Impact factor: 11.454

8.  Postintegrative gene silencing within the Sleeping Beauty transposition system.

Authors:  Brian S Garrison; Stephen R Yant; Jacob Giehm Mikkelsen; Mark A Kay
Journal:  Mol Cell Biol       Date:  2007-10-15       Impact factor: 4.272

9.  Site-directed transposon integration in human cells.

Authors:  Stephen R Yant; Yong Huang; Bassel Akache; Mark A Kay
Journal:  Nucleic Acids Res       Date:  2007-03-07       Impact factor: 16.971

Review 10.  Predicting preferential DNA vector insertion sites: implications for functional genomics and gene therapy.

Authors:  Christopher S Hackett; Aron M Geurts; Perry B Hackett
Journal:  Genome Biol       Date:  2007       Impact factor: 13.583
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  59 in total

1.  Adeno-Associated Virus-Based Gene Therapy for Lifelong Correction of Genetic Disease.

Authors:  Christian M Brommel; Ashley L Cooney; Patrick L Sinn
Journal:  Hum Gene Ther       Date:  2020-08-21       Impact factor: 5.695

Review 2.  Mesenchymal stem cell-based tumor-targeted gene therapy in gastrointestinal cancer.

Authors:  Qi Bao; Yue Zhao; Hanno Niess; Claudius Conrad; Bettina Schwarz; Karl-Walter Jauch; Ralf Huss; Peter J Nelson; Christiane J Bruns
Journal:  Stem Cells Dev       Date:  2012-06-26       Impact factor: 3.272

Review 3.  Manufacture of T cells using the Sleeping Beauty system to enforce expression of a CD19-specific chimeric antigen receptor.

Authors:  H Singh; J S E Moyes; M H Huls; L J N Cooper
Journal:  Cancer Gene Ther       Date:  2015-01-16       Impact factor: 5.987

4.  Transgene Expression in Dogs After Liver-Directed Hydrodynamic Delivery of Sleeping Beauty Transposons Using Balloon Catheters.

Authors:  Kendra A Hyland; Elena L Aronovich; Erik R Olson; Jason B Bell; Myra Urness Rusten; Roland Gunther; David W Hunter; Perry B Hackett; R Scott McIvor
Journal:  Hum Gene Ther       Date:  2017-04-19       Impact factor: 5.695

Review 5.  Gene targeting in ischemic heart disease and failure: translational and clinical studies.

Authors:  Shaina R Eckhouse; Jeffrey A Jones; Francis G Spinale
Journal:  Biochem Pharmacol       Date:  2012-08-28       Impact factor: 5.858

Review 6.  Non-viral vectors for gene-based therapy.

Authors:  Hao Yin; Rosemary L Kanasty; Ahmed A Eltoukhy; Arturo J Vegas; J Robert Dorkin; Daniel G Anderson
Journal:  Nat Rev Genet       Date:  2014-07-15       Impact factor: 53.242

Review 7.  A new approach to gene therapy using Sleeping Beauty to genetically modify clinical-grade T cells to target CD19.

Authors:  Harjeet Singh; Helen Huls; Partow Kebriaei; Laurence J N Cooper
Journal:  Immunol Rev       Date:  2014-01       Impact factor: 12.988

Review 8.  The potential of gene therapy approaches for the treatment of hemoglobinopathies: achievements and challenges.

Authors:  Michael A Goodman; Punam Malik
Journal:  Ther Adv Hematol       Date:  2016-06-25

Review 9.  Evaluating risks of insertional mutagenesis by DNA transposons in gene therapy.

Authors:  Perry B Hackett; David A Largaespada; Kirsten C Switzer; Laurence J N Cooper
Journal:  Transl Res       Date:  2013-01-10       Impact factor: 7.012

Review 10.  Gene transfer to the outflow tract.

Authors:  Yalong Dang; Ralitsa Loewen; Hardik A Parikh; Pritha Roy; Nils A Loewen
Journal:  Exp Eye Res       Date:  2016-04-27       Impact factor: 3.467
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