The University of Iowa Vector Core is integrated into multiple gene therapy projects directed at the study of human disease genes in vitro and in vivo. Vector Core staff are active participants in the development of gene transfer technologies based on multiple vector systems. Interactions between Vector Core staff and colleagues Paulson, Dauer, Gonzalez-Alegre, Kosik, Kotin, and Engelhardt allow for cross-fertilization of ideas, technical advancements, and innovations in vector design. In addition to the Project PIs and consultants, the Vector Core benefits from interactions with investigators in the Cardiovascular, Macular Degeneration, Cystic Fibrosis, and Gene Therapy Centers. The Vector Core facility's overall objective is to support investigators of the PPG in the use of gene transfer technologies. This includes consultation, development of appropriate shuttles, development of vectors most appropriate for culture studies or transduction of CNS cells in vivo, collaborative testing of vectors generated for function and purity, and finally routine preparation. The Vector Core staff and investigators are in close contact through all phases of vector design and generation. Thus, the Core serves as a research and development facility for gene transfer studies, and a service facility for routine vector preparations. Also importantly, the Vector Core allows Project PIs the opportunity to compare the beneficial and untoward effects of RNAi across different neurodegenerative disease models. The Vector Core's major service is to provide purified and concentrated preparations of recombinant adeno-associated virus (AAV), and lentivirus. This facility will also provide access to standard cell lines, expression plasmids, and stocks of recombinant reporter viruses. The main responsibilities of the Core will be: 1) Prepare recombinant vectors;2) Quality control;3) Vector dissemination;4) Maintain a database of vector stocks available for use;5) Catalogue plasmid database of expression vectors, develop new expression vectors as needed;6) Develop novel methods for virus production;7) Design and develop novel vectors.
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| Monteys, Alex Mas; Spengler, Ryan M; Dufour, Brett D et al. (2014) Single nucleotide seed modification restores in vivo tolerability of a toxic artificial miRNA sequence in the mouse brain. Nucleic Acids Res 42:13315-27 |
| Ramachandran, Pavitra S; Boudreau, Ryan L; Schaefer, Kellie A et al. (2014) Nonallele specific silencing of ataxin-7 improves disease phenotypes in a mouse model of SCA7. Mol Ther 22:1635-42 |
| Ramachandran, Pavitra S; Bhattarai, Sajag; Singh, Pratibha et al. (2014) RNA interference-based therapy for spinocerebellar ataxia type 7 retinal degeneration. PLoS One 9:e95362 |
| Lee, John H; Sowada, Matthew J; Boudreau, Ryan L et al. (2014) Rhes suppression enhances disease phenotypes in Huntington's disease mice. J Huntingtons Dis 3:65-71 |
| Boudreau, Ryan L; Jiang, Peng; Gilmore, Brian L et al. (2014) Transcriptome-wide discovery of microRNA binding sites in human brain. Neuron 81:294-305 |
| Rodríguez-Lebrón, Edgardo; Costa, Maria do Carmo; Costa, Maria doCarmo et al. (2013) Silencing mutant ATXN3 expression resolves molecular phenotypes in SCA3 transgenic mice. Mol Ther 21:1909-18 |
| Costa, Maria do Carmo; Luna-Cancalon, Katiuska; Fischer, Svetlana et al. (2013) Toward RNAi therapy for the polyglutamine disease Machado-Joseph disease. Mol Ther 21:1898-908 |
| Ramachandran, Pavitra S; Keiser, Megan S; Davidson, Beverly L (2013) Recent advances in RNA interference therapeutics for CNS diseases. Neurotherapeutics 10:473-85 |
| Boudreau, Ryan L; Spengler, Ryan M; Hylock, Ray H et al. (2013) siSPOTR: a tool for designing highly specific and potent siRNAs for human and mouse. Nucleic Acids Res 41:e9 |
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