Search Results - "Poirier, Amy E."

AAV Gene Therapy Utilizing Glycosylation-Independent Lysosomal Targeting Tagged GAA in the Hypoglossal Motor System of Pompe Mice by Doyle, Brendan M., Turner, Sara M.F., Sunshine, Michael D., Doerfler, Phillip A., Poirier, Amy E., Vaught, Lauren A., Jorgensen, Marda L., Falk, Darin J., Byrne, Barry J., Fuller, David D.

“…Pompe disease is caused by mutations in the gene encoding the lysosomal glycogen-metabolizing enzyme, acid-alpha glucosidase (GAA). Tongue myofibers and…”
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Striatal Readministration of rAAV Vectors Reveals an Immune Response Against AAV2 Capsids That Can Be Circumvented by Peden, Carmen S, Manfredsson, Fredric P, Reimsnider, Sharon K, Poirier, Amy E, Burger, Corinna, Muzyczka, Nicholas, Mandel, Ronald J

“…Recombinant adeno-associated virus (rAAV) expresses no viral genes after transduction. In addition, because the brain is relatively immunoprivileged,…”
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216. Long Term Portal Vein Administration of AAV-WPRE Vector Results in Increased Incidence of Neoplastic Disease and Hepatic Pathology by Embury, Jennifer E., Charron, Catherine C., Poirier, Amy E., Zori, Andreas, Carmichael, Russ, Flotte, Terry R., Laipis, Philip J.

“…Utilizing the Pahenu2 mouse model for phenylketonuria, we developed several expression vectors containing the Woodchuck Hepatitis Virus post-transcriptional…”
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Long‐term Delivery of “Low Dose” Repetitive Intermittent Hypoxia is Not Associated with Detectable Pathology by Gonzalez‐Rothi, Elisa Janine, Allen, Latoya A., Santiago‐Moreno, Juan, Ciesla, Marissa C., Asa, Zachary A., Smith, Kristin N., Tadjalli, Arash, Perim, Raphael, Santiago, Juliet V., Holland, Ashley E., Stefan, Kelsey A., Ross, Ashley, Satriotomo, Irawan, Kelly, Mia N., Simon, Alec K., Poirier, Amy E., Seven, Yasin B., Yarrow, Joshua F., Mitchell, Gordon S.

“…Repetitive acute intermittent hypoxia (rAIH) is a promising therapeutic strategy to induce spinal plasticity and improve respiratory and non‐respiratory…”
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101. Toxicology and Biodistribution Studies of a Recombinant Adeno-Associated Virus 2 (rAAV2) Alpha-1 Antitrypsin (AAT) Vector by Poirier, Amy E, Combee, Lynn A, Martino, Ashley T, Flotte, Terence R

“…A series of safety and biodistribution studies were undertaken to support the development of a muscle-based gene therapy system for AAT deficiency. AAT is the…”
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