Awakening gene therapy with Sleeping Beauty transposons

Jeffrey J. Essner; R. Scott McIvor; Perry B. Hackett

doi:10.1016/j.coph.2005.04.015

Awakening gene therapy with Sleeping Beauty transposons

Jeffrey J. Essner, R. Scott McIvor, Perry B. Hackett

Research output: Contribution to journal › Review article › peer-review

36 Scopus citations

Abstract

Sleeping Beauty transposons have the potential for use as chromosome-integrating vectors for non-viral gene therapy. Recent preclinical data from mouse models for human genetic disorders have shown efficacy for the Sleeping Beauty transposon system in the treatment of hemophilia, tyrosinemia type I, junctional epidermolysis bullosa and type 1 diabetes. Methods have also been developed to deliver Sleeping Beauty transposons to the lung, liver and tumors for treatments for cystic fibrosis, cardiovascular and metabolic diseases, and cancer. Recent studies characterizing site selection for integration and insertional mutagenesis indicate that the Sleeping Beauty transposon system may be a safer alternative than viral approaches for gene therapy.

Original language	English (US)
Pages (from-to)	513-519
Number of pages	7
Journal	Current Opinion in Pharmacology
Volume	5
Issue number	5 SPEC.ISS.
DOIs	https://doi.org/10.1016/j.coph.2005.04.015
State	Published - Oct 2005

Bibliographical note

Funding Information:
We thank Dr. Maura A McGrail for her thoughtful comments. The authors were supported by NIH grants P01 HD32652-07 (PBH and RSM) and R43 HL076908-01.

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10.1016/j.coph.2005.04.015

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abstract = "Sleeping Beauty transposons have the potential for use as chromosome-integrating vectors for non-viral gene therapy. Recent preclinical data from mouse models for human genetic disorders have shown efficacy for the Sleeping Beauty transposon system in the treatment of hemophilia, tyrosinemia type I, junctional epidermolysis bullosa and type 1 diabetes. Methods have also been developed to deliver Sleeping Beauty transposons to the lung, liver and tumors for treatments for cystic fibrosis, cardiovascular and metabolic diseases, and cancer. Recent studies characterizing site selection for integration and insertional mutagenesis indicate that the Sleeping Beauty transposon system may be a safer alternative than viral approaches for gene therapy.",

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Awakening gene therapy with Sleeping Beauty transposons

Abstract

Bibliographical note

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