We have ongoing projects that focus on gene therapy and genome editing in animals. For gene therapy we are improving the delivery and placement of Sleeping Beauty transposons in mouse and human genomes in order to improve their use as non-viral vectors. We use TALENs and other site-specific nucleases for precision genetic engineering (genome editing) to introduce specific sequences into animal genomes. We also use site-specific nucleases to engineer large animals to be hosts for human iPSC-derived tissues. We collaborate with academic partners (especially those in the Center for Genome Engineering, the UMN Stem Cell Institute, and the Masonic Cancer Center at UMN) as well as 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
Maiti, S., et al. (2013). Sleeping Beauty system to redirect T-cell specificity for human applications. J. Immunol. 36: 112-123.
Tan, W., D.F. Carlson, C.A. Lancto, D. Webster, P.B. Hackett, S.C. Fahrenkrug (2013) Efficient, non-meiotic introgression in livestock using custom endonucleases.
Carpenter, C.E., J. Schreifels, E.L. Aronovich, Carlson, D.F., P.B. Hackett, and I.V. Nesmelova (2014). Structure of Sleeping Beauty transposase binding to DNA. Prot. Sci. 23: 23-33
Hackett, P.B., T.K. Starr, and L.C. Cooper (2015). Risks of insertional mutagenesis by DNA transposons in cancer gene therapy. Transl. Med. Chapter 5, 65-84
Hackett, P.B., Fahrenkrug, S.C., Carlson, D.F. (2015). The promises and challenges of precision gene editing in animals of agricultural importance. IN Eaglesham, A. and Hardy, R.W.F, eds. New DNA-Editing Approaches: Methods, Applications and Policy for Agriculture. NABC Report 26, pp. 39-.
Hackett, P., Carroll, D. (2015). Regulatory hurdles for agriculture GMOs. Science 347: 1324.
Kebriae, P., et al. (2016) Phase I trials using Sleeping Beauty to generate CD19-specific T cells. J. Clin. Invest. 126: 3363-3376
Fong, Y., Hackett, P.B. (2017). Acceptance and Access to Gene Editing: Science and Our Obligations to Mankind. Mol. Ther. 25: 1-2
Hyland, K.A., et al. (2017). Transgene expression in dogs after liver-directed hydrodynamic delivery of Sleeping Beauty transposons using balloon catheters. Hum. Gene Ther. (in press)
Aronovich, E.L., et al. (2017). Treatment of a-L-iduronidase deficiency in dogs using the Sleeping Beauty transposon system. Hum. Gene Ther. (in press)
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.