Awakening gene therapy with Sleeping Beauty transposons

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

Research output: Contribution to journalReview articlepeer-review

35 Scopus citations


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 languageEnglish (US)
Pages (from-to)513-519
Number of pages7
JournalCurrent Opinion in Pharmacology
Issue number5 SPEC.ISS.
StatePublished - 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.

Copyright 2018 Elsevier B.V., All rights reserved.

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