title: Awakening gene therapy with Sleeping Beauty transposons.
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.
authors.affiliation: Discovery Genomics Inc, Minneapolis, Minnesota 55413, USA. jeffreye@discoverygenomics.net
authors.forename: Jeffrey J
authors.initials: JJ
authors.lastname: Essner
authors.forename: R Scott
authors.initials: RS
authors.lastname: McIvor
authors.forename: Perry B
authors.initials: PB
authors.lastname: Hackett
mesh_terms: D000818:Animals; D004251:DNA Transposable Elements / Q000235:genetics*; D015316:Genetic Therapy / Q000009:adverse effects / Q000379:methods*; D005822:Genetic Vectors / Q000009:adverse effects / Q000235:genetics*; D006801:Humans; D012449:Safety; D019895:Transposases / Q000235:genetics / Q000378:metabolism
chemical_list: D004251:DNA Transposable Elements; D019895:Transposases; C494471:sleeping beauty transposase, human