Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of N... von Belur, Lalitha R, Temme, Alexa, Podetz-Pedersen, Kelly M, Riedl, Maureen, Vulchanova, Lucy, Robinson, Nicholas, Hanson, Leah R, Kozarsky, Karen F, Orchard, Paul J, Frey, 2nd, William H, Low, Walter C, McIvor, R Scott

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Biodistribution of adeno-associated virus serotype 9 (AAV9) vector after intrathecal and intravenous delivery in mouse von Schuster, Daniel J, Dykstra, Jaclyn A, Riedl, Maureen S, Kitto, Kelley F, Belur, Lalitha R, McIvor, R Scott, Elde, Robert P, Fairbanks, Carolyn A, Vulchanova, Lucy

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Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolys... von Belur, Lalitha R., Romero, Megan, Lee, Junggu, Podetz-Pedersen, Kelly M., Nan, Zhenhong, Riedl, Maureen S., Vulchanova, Lucy, Kitto, Kelley F., Fairbanks, Carolyn A., Kozarsky, Karen F., Orchard, Paul J., Frey, William H., Low, Walter C., McIvor, R. Scott

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Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I von Wolf, Daniel A, Lenander, Andrew W, Nan, Zhenhong, Belur, Lalitha R, Whitley, Chester B, Gupta, Pankaj, Low, Walter C, McIvor, R. Scott

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Differential Adeno-Associated Virus Mediated Gene Transfer to Sensory Neurons following Intrathecal Delivery by Direct Lumbar Puncture von Vulchanova, Lucy, Schuster, Daniel J, Belur, Lalitha R, Riedl, Maureen S, Podetz-Pedersen, Kelly M, Kitto, Kelley F, Wilcox, George L, McIvor, R Scott, Fairbanks, Carolyn A

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Preferential delivery of the Sleeping Beauty transposon system to livers of mice by hydrodynamic injection von Hackett, Perry B, Bell, Jason B, Podetz-Pedersen, Kelly M, Aronovich, Elena L, Belur, Lalitha R, McIvor, R Scott

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Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice von Smith, Miles C., Belur, Lalitha R., Karlen, Andrea D., Erlanson, Olivia, Furcich, Justin, Lund, Troy C., Seelig, Davis, Kitto, Kelley F., Fairbanks, Carolyn A., Kim, Kwi Hye, Buss, Nick, McIvor, R. Scott

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Non-invasive intravenous administration of AAV9 transducing iduronate sulfatase leads to global metabolic correction and prevention of neurologic deficits in a mouse model of Hunte... von Laoharawee, Kanut, Podetz-Pedersen, Kelly M., Nguyen, Tam T., Singh, Sajya M., Smith, Miles C., Belur, Lalitha R., Low, Walter C., Kozarsky, Karen F., McIvor, R. Scott

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Adeno-associated virus-mediated gene transfer of arginine decarboxylase to the central nervous system prevents opioid analgesic tolerance von Churchill, Caroline C, Peterson, Cristina D, Kitto, Kelley F, Pflepsen, Kelsey R, Belur, Lalitha R, McIvor, R Scott, Vulchanova, Lucy, Wilcox, George L, Fairbanks, Carolyn A

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Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses von Aronovich, Elena L., Bell, Jason B., Belur, Lalitha R., Gunther, Roland, Koniar, Brenda, Erickson, David C. C., Schachern, Patricia A., Matise, Ilze, McIvor, R. Scott, Whitley, Chester B., Hackett, Perry B.

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Inhibition of angiogenesis and suppression of colorectal cancer metastatic to the liver using the Sleeping Beauty Transposon System von Belur, Lalitha R, Podetz-Pedersen, Kelly M, Sorenson, Brent S, Hsu, Alice H, Parker, Josh B, Carlson, Cathy S, Saltzman, Daniel A, Ramakrishnan, S, McIvor, R Scott

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Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS After the Development of Cognitive Dysfunction von Podetz-Pedersen, Kelly M, Laoharawee, Kanut, Singh, Sajya, Nguyen, Tam T, Smith, Miles C, Temme, Alexa, Kozarsky, Karen, McIvor, R Scott, Belur, Lalitha R

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Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of Ex Vivo Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells von Smith, Miles C, Belur, Lalitha R, Karlen, Andrea D, Erlanson, Olivia, Podetz-Pedersen, Kelly M, McKenzie, Jessica, Detellis, Jenn, Gagnidze, Khatuna, Parsons, Geoffrey, Robinson, Nicholas, Labarre, Shelby, Shah, Saumil, Furcich, Justin, Lund, Troy C, Tsai, Hsing-Chen, McIvor, R Scott, Bonner, Melissa

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Long-term reversal of chronic pain behavior in rodents through elevation of spinal agmatine von Peterson, Cristina D., Waataja, Jonathan J., Kitto, Kelley F., Erb, Samuel J., Verma, Harsha, Schuster, Daniel J., Churchill, Caroline C., Riedl, Maureen S., Belur, Lalitha R., Wolf, Daniel A., McIvor, R. Scott, Vulchanova, Lucy, Wilcox, George L., Fairbanks, Carolyn A.

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Generation and characterization of an immunodeficient mouse model of mucopolysaccharidosis type II von Smith, Miles C., Belur, Lalitha R., Karlen, Andrea D., Podetz-Pedersen, Kelly, Erlanson, Olivia, Laoharawee, Kanut, Furcich, Justin, Lund, Troy C., You, Yun, Seelig, Davis, Webber, Beau R., McIvor, R. Scott

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Intravenous delivery for treatment of mucopolysaccharidosis type I: A comparison of AAV serotypes 9 and rh10 von Belur, Lalitha R., Podetz-Pedersen, Kelly M., Tran, Thuy An, Mesick, Joshua A., Singh, Nathaniel M., Riedl, Maureen, Vulchanova, Lucy, Kozarsky, Karen F., McIvor, R. Scott

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Systemic Correction of Storage Disease in MPS I NOD/SCID Mice Using the Sleeping Beauty Transposon System von Aronovich, Elena L, Bell, Jason B, Khan, Shaukat A, Belur, Lalitha R, Gunther, Roland, Koniar, Brenda, Schachern, Patricia A, Parker, Josh B, Carlson, Cathy S, Whitley, Chester B, McIvor, R Scott, Gupta, Pankaj, Hackett, Perry B

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Efficient and Stable Transgene Expression in Human Embryonic Stem Cells Using Transposon‐Mediated Gene Transfer von Wilber, Andrew, Linehan, Jonathan L., Tian, Xinghui, Woll, Petter S., Morris, Julie K., Belur, Lalitha R., McIvor, R. Scott, Kaufman, Dan S.

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Supraspinal gene transfer by intrathecal adeno-associated virus serotype 5 von Schuster, Daniel J, Belur, Lalitha R, Riedl, Maureen S, Schnell, Stephen A, Podetz-Pedersen, Kelly M, Kitto, Kelley F, McIvor, R Scott, Vulchanova, Lucy, Fairbanks, Carolyn A

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Lung-directed gene therapy in mice using the nonviral Sleeping Beauty transposon system von Belur, Lalitha R, McIvor, R Scott, Podetz-Pedersen, Kelly, Frandsen, Joel

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