Search Results - "HACKETT, Perry B"

Efficient nonmeiotic allele introgression in livestock using custom endonucleases by Tan, Wenfang, Carlson, Daniel F., Lancto, Cheryl A., Garbe, John R., Webster, Dennis A., Hackett, Perry B., Fahrenkrug, Scott C.

“…We have expanded the livestock gene editing toolbox to include transcription activator-like (TAL) effector nuclease (TALEN)- and clustered regularly…”
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Mapping brain networks in MPS I mice and their restoration following gene therapy by Zhu, Wei, Ou, Li, Zhang, Lin, Clark, Isaac H., Zhang, Ying, Zhu, Xiao-Hong, Whitley, Chester B., Hackett, Perry B., Low, Walter C., Chen, Wei

“…Mucopolysaccharidosis type I (MPS I) is an inherited lysosomal disorder that causes syndromes characterized by physiological dysfunction in many organs and…”
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Glycopolycation–DNA Polyplex Formulation N/P Ratio Affects Stability, Hemocompatibility, and in Vivo Biodistribution by Phillips, Haley R, Tolstyka, Zachary P, Hall, Bryan C, Hexum, Joseph K, Hackett, Perry B, Reineke, Theresa M

“…Genome editing therapies hold great promise for the cure of monogenic and other diseases; however, the application of nonviral gene delivery methods is limited…”
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Harnessing a high cargo-capacity transposon for genetic applications in vertebrates by Balciunas, Darius, Wangensteen, Kirk J, Wilber, Andrew, Bell, Jason, Geurts, Aron, Sivasubbu, Sridhar, Wang, Xin, Hackett, Perry B, Largaespada, David A, McIvor, R Scott, Ekker, Stephen C

“…Viruses and transposons are efficient tools for permanently delivering foreign DNA into vertebrate genomes but exhibit diminished activity when cargo exceeds 8…”
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Moesin1 and Ve-cadherin are required in endothelial cells during in vivo tubulogenesis by Wang, Ying, Kaiser, Mark S, Larson, Jon D, Nasevicius, Aidas, Clark, Karl J, Wadman, Shannon A, Roberg-Perez, Sharon E, Ekker, Stephen C, Hackett, Perry B, McGrail, Maura, Essner, Jeffrey J

“…Endothelial tubulogenesis is a crucial step in the formation of functional blood vessels during angiogenesis and vasculogenesis. Here, we use in vivo imaging…”
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Targeting DNA With Fingers and TALENs by Carlson, Daniel F, Fahrenkrug, Scott C, Hackett, Perry B

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Evaluating risks of insertional mutagenesis by DNA transposons in gene therapy by Hackett, Perry B, Largaespada, David A, Switzer, Kirsten C, Cooper, Laurence J.N

“…Investigational therapy can be successfully undertaken using viral- and nonviral-mediated ex vivo gene transfer. Indeed, recent clinical trials have…”
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Engineered Swine Models of Cancer by Watson, Adrienne L, Carlson, Daniel F, Largaespada, David A, Hackett, Perry B, Fahrenkrug, Scott C

“…Over the past decade, the technology to engineer genetically modified swine has seen many advancements, and because their physiology is remarkably similar to…”
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Preferential delivery of the Sleeping Beauty transposon system to livers of mice by hydrodynamic injection by Hackett, Perry B, Bell, Jason B, Podetz-Pedersen, Kelly M, Aronovich, Elena L, Belur, Lalitha R, McIvor, R Scott

“…Nonviral, DNA-mediated gene transfer is an alternative to viral delivery systems for expressing new genes in cells and tissues. The Sleeping Beauty (SB)…”
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Gene therapy for neurologic manifestations of mucopolysaccharidoses by Wolf, Daniel A, Banerjee, Sharbani, Hackett, Perry B, Whitley, Chester B, McIvor, R Scott, Low, Walter C

“…Mucopolysaccharidoses (MPS) are a family of lysosomal disorders caused by mutations in genes that encode enzymes involved in the catabolism of…”
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Efficacy and safety of Sleeping Beauty transposon-mediated gene transfer in preclinical animal studies by Hackett, Jr, Perry B, Aronovich, Elena L, Hunter, David, Urness, Myra, Bell, Jason B, Kass, Steven J, Cooper, Laurence J N, McIvor, Scott

“…Sleeping Beauty (SB) transposons have been effective in delivering therapeutic genes to treat certain diseases in mice. Hydrodynamic gene delivery of…”
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Molecular Reconstruction of Sleeping Beauty, a Tc1-like Transposon from Fish, and Its Transposition in Human Cells by Ivics, Zoltán, Hackett, Perry B, Plasterk, Ronald H, Izsvák, Zsuzsanna

“…Members of the Tc1/mariner superfamily of transposons isolated from fish appear to be transpositionally inactive due to the accumulation of mutations…”
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Quantitative analysis of α-L-iduronidase expression in immunocompetent mice treated with the Sleeping Beauty transposon system by Aronovich, Elena L, Hall, Bryan C, Bell, Jason B, McIvor, R Scott, Hackett, Perry B

“…The Sleeping Beauty transposon system, a non-viral, integrating vector that can deliver the alpha-L-iduronidase-encoding gene, is efficient in correcting…”
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Trehalose-Based Block Copolycations Promote Polyplex Stabilization for Lyophilization and in Vivo pDNA Delivery by Tolstyka, Zachary P, Phillips, Haley, Cortez, Mallory, Wu, Yaoying, Ingle, Nilesh, Bell, Jason B, Hackett, Perry B, Reineke, Theresa M

“…The development and thorough characterization of nonviral delivery agents for nucleic acid and genome editing therapies are of high interest to the field of…”
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Structure–Function Analysis of the Inverted Terminal Repeats of the Sleeping Beauty Transposon by Cui, Zongbin, Geurts, Aron M., Liu, Geyi, Kaufman, Christopher D., Hackett, Perry B.

“…Translocation of Sleeping Beauty (SB) transposon requires specific binding of SB transposase to inverted terminal repeats (ITRs) of about 230bp at each end of…”
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Effective gene trapping mediated by Sleeping Beauty transposon by Song, Guili, Li, Qing, Long, Yong, Gu, Qilin, Hackett, Perry B, Cui, Zongbin

“…Gene trapping is a high-throughput approach to elucidate gene functions by disrupting and recapitulating expression of genes in a target genome. A number of…”
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Strategies for selection marker-free swine transgenesis using the Sleeping Beauty transposon system by Carlson, Daniel F, Garbe, John R, Tan, Wenfang, Martin, Mike J, Dobrinsky, John R, Hackett, Perry B, Clark, Karl J, Fahrenkrug, Scott C

“…Swine transgenesis by pronuclear injection or cloning has traditionally relied on illegitimate recombination of DNA into the pig genome. This often results in…”
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Asexual reproduction for improved livestock breeding by Fan, Ziyao, Gu, Taotao, Hackett, Perry B., Li, Kui

“…As natural environments deteriorate, genetic improvements to agricultural animals will be required to ensure global food security. Improving livestock…”
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Predicting preferential DNA vector insertion sites: implications for functional genomics and gene therapy by Hackett, Christopher S, Geurts, Aron M, Hackett, Perry B

“…Viral and transposon vectors have been employed in gene therapy as well as functional genomics studies. However, the goals of gene therapy and functional…”
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Phenotypic correction and long-term expression of factor VIII in hemophilic mice by immunotolerization and nonviral gene transfer using the Sleeping Beauty transposon system by Ohlfest, John R., Frandsen, Joel L., Fritz, Sabine, Lobitz, Paul D., Perkinson, Scott G., Clark, Karl J., Nelsestuen, Gary, Key, Nigel S., McIvor, R.Scott, Hackett, Perry B., Largaespada, David A.

“…Hemophilia A is a lead candidate for treatment by gene therapy because small increments in the missing secreted protein product, coagulation factor VIII…”
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