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 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 a biotech company that is a spinout from our lab (Recombinetics, Inc.) in order to deliver the greatest impact of our work to society.
Fong, Y., Hackett, P.B. (2017). Acceptance and Access to Gene Editing: Science and Our Obligations to Mankind. Mol. Ther. 25: 1-2
FHyland, K.A., et al. (2017). Transgene expression in dogs after liver-directed hydrodynamic delivery of Sleeping Beauty transposons using balloon catheters. Hum. Gene Ther. 28: 541-550
FAronovich, E.L., et al. (2017). Treatment of a-L-iduronidase deficiency in dogs using the Sleeping Beauty transposon system. Hum. Gene Ther. 28: 551-564Hackett, 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
FKebriae, P., et al. (2016) Phase I trials using Sleeping Beauty to generate CD19-specific T cells. J. Clin. Invest. 126: 3363-3376.
FWatson AL, Carlson DF, Largaespada DA, Hackett PB, Fahrenkrug SC. (2016). Engineered swine models of cancer. Front. Genet. 7: 78.
FDhande YK, et al. (2016). N-acetylgalactosamine block-co-polycations form stable complexes with plasmids and promote liver-targeted delivery. Biomacromolecules 17: 830-840.
FTolstyka, ZP, et al. (2016) Trehalose-based block copolymers for in vitro and in vivo non-viral DNA delivery. Biomaterials ACS Biomat. Sci. Eng. 2: 43-55.
FHackett, 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. 39-52.
FHackett, P., Carroll, D. (2015). Regulatory hurdles for agriculture GMOs. Science 347: 1324.
FAronovich, EA, Hackett, PB (2015). Gene therapy on both sides of the blood-brain barrier. Mol. Genet. Metab. 114: 83-93.
FWolf, DA, Banerjee, S, Hackett, PB, Whitley, CB, McIvor, RS, Low, WC (2015). Gene therapy for neurologic manifestations of mucopolysaccharidoses. Expert Opin. Drug. Deliv. 12: 283-296
FHackett, PB, Aronovich, EL. (2014) Rational design for enhanced gene therapy with DNA transposons (2014). Mol. Ther. 22: 1575-1577.
FHackett, PB, Somia, N. (2014). Delivery of the second revolution in site-specific endonucleases. eLIFE 3: e02904
FCarpenter, 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