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Start Over Author "Scheule, Ronald" Journal molecular therapy
20 results on '"Scheule, Ronald"'

1. Systemic Administration of AAV8-α-galactosidase A Induces Humoral Tolerance in Nonhuman Primates Despite Low Hepatic Expression

Author

Nietupski, Jennifer B, primary, Hurlbut, Gregory D, additional, Ziegler, Robin J, additional, Chu, Qiuming, additional, Hodges, Bradley L, additional, Ashe, Karen M, additional, Bree, Mark, additional, Cheng, Seng H, additional, Gregory, Richard J, additional, Marshall, John, additional, and Scheule, Ronald K, additional

Published
2011
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3. Preexisting Immunity and Low Expression in Primates Highlight Translational Challenges for Liver-directed AAV8-mediated Gene Therapy

Author

Hurlbut, Gregory D, primary, Ziegler, Robin J, additional, Nietupski, Jennifer B, additional, Foley, Joseph W, additional, Woodworth, Lisa A, additional, Meyers, Elizabeth, additional, Bercury, Scott D, additional, Pande, Nilesh N, additional, Souza, David W, additional, Bree, Mark P, additional, Lukason, Michael J, additional, Marshall, John, additional, Cheng, Seng H, additional, and Scheule, Ronald K, additional

Published
2010
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6. Timing of Therapeutic Intervention Determines Functional and Survival Outcomes in a Mouse Model of Late Infantile Batten Disease

Author

Cabrera-Salazar, Mario A, primary, Roskelley, Eric M, additional, Bu, Jie, additional, Hodges, Bradley L, additional, Yew, Nelson, additional, Dodge, James C, additional, Shihabuddin, Lamya S, additional, Sohar, Istvan, additional, Sleat, David E, additional, Scheule, Ronald K, additional, Davidson, Beverly L, additional, Cheng, Seng H, additional, Lobel, Peter, additional, and Passini, Marco A, additional

Published
2007
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7. 475. SeV-Mediated Gene Transfer after Three Re-Administrations in Mice and Sheep

Author

Griesenbach, Uta, primary, McLachlan, Gerry, additional, Somerton, Lucinda, additional, Collie, David, additional, Inoue, Makoto, additional, Hironaka, Takashi, additional, Cheng, Seng, additional, Scheule, Ronald, additional, Owaki, Toshiyuki, additional, Baker, Alison, additional, Tennant, Peter, additional, Hasegawa, Mamoru, additional, and Alton, Eric W.F.W., additional

Published
2006
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10. 695. Low-Frequency Ultrasound Increases Non- Viral Lung Gene Transfer

Author

Xenariou, Stefania, primary, Liang, Hai-Dong, additional, Griesenbach, Uta, additional, Zhu, Jie, additional, Farley, Raymond, additional, Somerton, Lucinda, additional, Singh, Charanjit, additional, Jeffery, Peter K., additional, Scheule, Ronald K., additional, Cheng, Seng H., additional, Geddes, Duncan M., additional, Blomley, Martin, additional, and Alton, Eric W.F.W., additional

Published
2006
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18. Induction of Immune Tolerance to a Therapeutic Protein by Intrathymic Gene Delivery.

Author

Qiuming Chu, Moreland, Rodney J., Lan Gao, Taylor, Kristin M., Meyers, Elizabeth, Cheng, Seng H., and Scheule, Ronald K.

Subjects
*IMMUNOLOGICAL tolerance, *IMMUNE response, *PROTEINS, *GENETIC disorders, *T cells, *MESSENGER RNA
Abstract

The efficacy of recombinant enzyme therapy for genetic diseases is limited in some patients by the generation of a humoral immune response to the therapeutic protein. Inducing immune tolerance to the protein prior to treatment has the potential to increase therapeutic efficacy. Using an AAV8 vector encoding human acid α-glucosidase (hGAA), we have evaluated direct intrathymic injection for inducing tolerance. We have also compared the final tolerogenic states achieved by intrathymic and intravenous injection. Intrathymic vector delivery induced tolerance equivalent to that generated by intravenous delivery, but at a 25-fold lower dose, the thymic hGAA expression level was 10,000-fold lower than the liver expression necessary for systemic tolerance induction. Splenic regulatory T cells (Tregs) were apparent after delivery by both routes, but with different phenotypes. Intrathymic delivery resulted in Tregs with higher FoxP3, TGFβ, and IL-10 mRNA levels. These differences may account for the differences noted in splenic T cells, where only intravenous delivery appeared to inhibit their activation. Our results imply that different mechanisms may be operating to generate immune tolerance by intrathymic and intravenous delivery of an AAV vector, and suggest that the intrathymic route may hold promise for decreasing the humoral immune response to therapeutic proteins in genetic disease indications. [ABSTRACT FROM AUTHOR]

Published
2010
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19. 531. Balloon Catheter-Mediated Hepatic Vein Delivery of a Viral Vector Mitigates Neutralization by Anti-Viral Antibodies and Results in Efficient Transduction of Rabbit Liver

Author

Hodges, Bradley L., Taylor, Kristin M., Chu, Qiuming, Scull, Samantha E., Serriello, Rebecca G., Anderson, Scott C., and Scheule, Ronald K.

Subjects
*HEPATIC veins, *GENETIC transformation
Abstract

An abstract of the article "Balloon Catheter-Mediated Hepatic Vein Delivery of a Viral Vector Mitigates Neutralization by Anti-Viral Antibodies and Results in Efficient Transduction of Rabbit Liver," by Bradley L. Hodgesand colleagues is presented.

Published
2005
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20. 695. Low-Frequency Ultrasound Increases Non-Viral Lung Gene Transfer.

Author

Stefania Xenariou, Hai-Dong Liang, Griesenbach, Uta, Jie Zhu, Farley, Raymond, Somerton, Lucinda, Singh, Charanjit, Jeffery, Peter K., Scheule, Ronald K., Cheng, Seng H., Geddes, Duncan M., Blomley, Martin, and Alton, Eric W. F. W.

Subjects
*GENETIC transformation, *MOBILE genetic elements, *CELL membranes, *LUNGS, *MICROBIAL genetics
Abstract

We have previously shown that high-frequency (1 MHz) ultrasound (U/S) increases naked plasmid DNA (pDNA) gene transfer in the mouse lung and that this effect requires microbubbles (Optison) to be present in the formulation. Here, we assessed low-frequency (30–35 kHz) U/S, in an attempt to further increase non- viral gene delivery to the lung in vivo. The potential advantages of lowering U/S frequency include: a) increased cavitation, the mechanism responsible for the formation of transient pores on cell membranes and b) enhanced penetration of U/S energy into the lung, by reducing wave scattering and heat losses. Cationic lipid GL67 (GL67)/pDNA (80 μg/100 μl), polyethylenimine (PEI)/pDNA (20 μg/100 μl) and naked pDNA (100 μg/100 μl) were delivered to Balb/ c mice via intranasal instillation. Low-frequency U/S (30–35 kHz, 0.07 or 0.1 Mpa, 10 min) was then applied onto the chest and back. Sonoporation did not enhance GL67/pDNA and PEI/pDNA gene transfer. However, U/S exposure at 0.1 Mpa, but not at 0.07 Mpa, increased naked pDNA transfection approximately 4-fold (pDNA: 5.9±1.2 RLU/mg protein, pDNA + 0.1 Mpa: 22.6±3.8 RLU/mg protein, p<0.05, n=7–8). Importantly, low-frequency U/S increased lung gene transfer in the absence of microbubbles. Addition of Optison did not further enhance this effect, due to its inhibitory effects on non-viral lung gene transfer (pDNA + Optison: 0.4±0.1 RLU/mg protein, pDNA + Optison + 0.1 Mpa: 2.5±0.7 RLU/mg protein, n=7–8). Haemorrhage, a common U/S-induced side-effect, was also assessed and increased with pressure in a dose-dependent manner. Interestingly, haemorrhage occurred throughout the lung, indicating that U/S may penetrate deep into the tissue. We have, thus, established for the first time that low-frequency sonoporation can enhance non-viral gene delivery to the mouse lung in vivo.Molecular Therapy (2006) 13, S269–S269; doi: 10.1016/j.ymthe.2006.08.773 [ABSTRACT FROM AUTHOR]

Published
2006
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