We have ongoing projects that focus on improving the delivery and placement of Sleeping Beauty transposons in mouse and human genomes in order to improve their use as non-viral vectors for human gene therapy. We use TALENs and other site-specific nucleases as well as RecA for specific placement of expression cassettes in genomes. We also use site-specific nucleases to engineer large animals to be hosts for human tissues. We collaborate with academic partners (especially those in the Center for Genome Engineering and the Masonic Cancer Center at UMN) and with two biotech companies that are spinouts from our lab and based in the Twin Cities (Discovery Genomics, Inc. and Recombinetics, Inc.) in order to deliver the greatest impact of our work to society.
Selected Publications (Pubmed Search)
Aronovich, EA, Hackett, PB (2015). Gene therapy on both sides of the blood-brain barrier. Mol. Genet. Metab. 114: 83-93.
Wolf, DA, Banerjee, S, Hackett, PB, Whitley, CB, McIvor, RS, Low, WC (2015). Gene therapy for neurologic manifestations mucopolysaccharidoses. Expert Opin. Drug. Deliv. 12: 283-296
Hackett, PB, Aronovich, EL. (2014) Rational design for enhanced gene therapy with DNA transposons (2014). Mol. Ther. 22: 1575-1577.
Hackett, PB, Somia, N. (2014). Delivery of the second revolution in site-specific endonucleases. eLIFE 3: e02904
Carpenter, CE, Schreifels, J, Aronovich, EL, Carlson, DF, Hackett, PB,. Nesmelova, IV. (2014). Structure of Sleeping Beauty transposase binding to DNA. Prot. Sci. 23: 23-33
Carlson, DF. Tan, W, Hackett, PB, Fahrenkrug, SC. (2013) Efficient, non-meiotic introgression in livestock using custom endonucleases. Reprod. Fertil. Dev. 26:74-82.
Aronovich, EL, Hall, BC, Bell, JB, McIvor, RS, Hackett, PB (2013) Factors affecting maintenance of Sleeping Beauty transposon-mediated expression of human alpha-L-iduronidase. PLoS One 8: e78161
Tan, W, Carlson, DF, Lancto, CA, Webster, D, Hackett, PB, Fahrenkrug, SC. (2013) Efficient, non-meiotic introgression in livestock using custom endonucleases. Proc. Natl. Acad. Sci. USA 110: 16526-16531.