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

Elena L. Aronovich; R. Scott McIvor; Perry B. Hackett

doi:10.1093/hmg/ddr140

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

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

Research output: Contribution to journal › Article › peer-review

117 Scopus citations

Abstract

Over the past decade, the Sleeping Beauty (SB) transposon system has been developed as the leading nonviral 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.

Original language	English (US)
Article number	ddr140
Pages (from-to)	R14-R20
Journal	Human molecular genetics
Volume	20
DOIs	https://doi.org/10.1093/hmg/ddr140
State	Published - Apr 2011

Bibliographical note

Funding Information:
We acknowledge the financial support of NIH grant 1R01DK082516.

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

Access

10.1093/hmg/ddr140

OpenUrl availability

Full text

Cite this

@article{7319e46c41bd4db6a75d358e6276309d,

title = "The Sleeping Beauty transposon system: A non-viral vector for gene therapy",

abstract = "Over the past decade, the Sleeping Beauty (SB) transposon system has been developed as the leading nonviral 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.",

author = "Aronovich, {Elena L.} and McIvor, {R. Scott} and Hackett, {Perry B.}",

note = "Funding Information: We acknowledge the financial support of NIH grant 1R01DK082516.",

year = "2011",

month = apr,

doi = "10.1093/hmg/ddr140",

language = "English (US)",

volume = "20",

pages = "R14--R20",

journal = "Human molecular genetics",

issn = "0964-6906",

publisher = "Oxford University Press",

}

TY - JOUR

T1 - The Sleeping Beauty transposon system

T2 - A non-viral vector for gene therapy

AU - Aronovich, Elena L.

AU - McIvor, R. Scott

AU - Hackett, Perry B.

N1 - Funding Information: We acknowledge the financial support of NIH grant 1R01DK082516.

PY - 2011/4

Y1 - 2011/4

N2 - Over the past decade, the Sleeping Beauty (SB) transposon system has been developed as the leading nonviral 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.

AB - Over the past decade, the Sleeping Beauty (SB) transposon system has been developed as the leading nonviral 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.

UR - http://www.scopus.com/inward/record.url?scp=79955990614&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=79955990614&partnerID=8YFLogxK

U2 - 10.1093/hmg/ddr140

DO - 10.1093/hmg/ddr140

M3 - Article

C2 - 21459777

AN - SCOPUS:79955990614

SN - 0964-6906

VL - 20

SP - R14-R20

JO - Human molecular genetics

JF - Human molecular genetics

M1 - ddr140

ER -

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

Abstract

Bibliographical note

UN SDGs

Access

OpenUrl availability

Other files and links

Fingerprint

Cite this