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3. Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus Monkeys

Author

Hordeaux, Juliette, Hinderer, Christian, Buza, Elizabeth L, Louboutin, Jean-Pierre, Jahan, Tahsin, Bell, Peter, Chichester, Jessica A, Tarantal, Alice F, and Wilson, James M

Subjects
Medical Biotechnology, Biomedical and Clinical Sciences, Rare Diseases, Brain Disorders, Genetics, Mucopolysaccharidoses (MPS), Pediatric, Biotechnology, Gene Therapy, Neurosciences, 5.2 Cellular and gene therapies, Neurological, Animals, Dependovirus, Female, Ganglia, Spinal, Gene Expression, Gene Transfer Techniques, Genetic Therapy, Genetic Vectors, Humans, Iduronidase, Immunohistochemistry, Injections, Spinal, Macaca mulatta, Neurons, Organ Specificity, Promoter Regions, Genetic, Serogroup, Tissue Distribution, Transgenes, AAV9, intrathecal, MPS I, infant, rhesus monkey, Clinical Sciences, Medical biotechnology
Abstract

Many neuropathic diseases cause early, irreversible neurologic deterioration, which warrants therapeutic intervention during the first months of life. In the case of mucopolysaccharidosis type I, a recessive lysosomal storage disorder that results from a deficiency of the lysosomal enzyme α-l-iduronidase (IDUA), one of the most promising treatment approaches is to restore enzyme expression through gene therapy. Specifically, administering pantropic adeno-associated virus (AAV) encoding IDUA into the cerebrospinal fluid (CSF) via suboccipital administration has demonstrated remarkable efficacy in large animals. Preclinical safety studies conducted in adult nonhuman primates supported a positive risk-benefit profile of the procedure while highlighting potential subclinical toxicity to primary sensory neurons located in the dorsal root ganglia (DRG). This study investigated the long-term performance of intrathecal cervical AAV serotype 9 gene transfer of human IDUA administered to 1-month-old rhesus monkeys (N = 4) with half of the animals tolerized to the human transgene at birth via systemic administration of an AAV serotype 8 vector expressing human IDUA from the liver. Sustained expression of the transgene for almost 4 years is reported in all animals. Transduced cells were primarily pyramidal neurons in the cortex and hippocampus, Purkinje cells in the cerebellum, lower motor neurons, and DRG neurons. Both tolerized and non-tolerized animals were robust and maintained transgene expression as measured by immunohistochemical analysis of brain tissue. However, the presence of antibodies in the non-tolerized animals led to a loss of measurable levels of secreted enzyme in the CSF. These results support the safety and efficiency of treating neonatal rhesus monkeys with AAV serotype 9 gene therapy delivered into the CSF.

Published
2019

13. Supplementary Figure Legends from Systematic In Vivo Inactivation of Chromatin-Regulating Enzymes Identifies Setd2 as a Potent Tumor Suppressor in Lung Adenocarcinoma

Author

Walter, David M., primary, Venancio, Olivia S., primary, Buza, Elizabeth L., primary, Tobias, John W., primary, Deshpande, Charuhas, primary, Gudiel, A. Andrea, primary, Kim-Kiselak, Caroline, primary, Cicchini, Michelle, primary, Yates, Travis J., primary, and Feldser, David M., primary

Published
2023
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14. Supplementary Table 1 from Systematic In Vivo Inactivation of Chromatin-Regulating Enzymes Identifies Setd2 as a Potent Tumor Suppressor in Lung Adenocarcinoma

Author

Walter, David M., primary, Venancio, Olivia S., primary, Buza, Elizabeth L., primary, Tobias, John W., primary, Deshpande, Charuhas, primary, Gudiel, A. Andrea, primary, Kim-Kiselak, Caroline, primary, Cicchini, Michelle, primary, Yates, Travis J., primary, and Feldser, David M., primary

Published
2023
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15. Supplementary Table 3 from Systematic In Vivo Inactivation of Chromatin-Regulating Enzymes Identifies Setd2 as a Potent Tumor Suppressor in Lung Adenocarcinoma

Author

Walter, David M., primary, Venancio, Olivia S., primary, Buza, Elizabeth L., primary, Tobias, John W., primary, Deshpande, Charuhas, primary, Gudiel, A. Andrea, primary, Kim-Kiselak, Caroline, primary, Cicchini, Michelle, primary, Yates, Travis J., primary, and Feldser, David M., primary

Published
2023
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16. Supplementary Table 2 from Systematic In Vivo Inactivation of Chromatin-Regulating Enzymes Identifies Setd2 as a Potent Tumor Suppressor in Lung Adenocarcinoma

Author

Walter, David M., primary, Venancio, Olivia S., primary, Buza, Elizabeth L., primary, Tobias, John W., primary, Deshpande, Charuhas, primary, Gudiel, A. Andrea, primary, Kim-Kiselak, Caroline, primary, Cicchini, Michelle, primary, Yates, Travis J., primary, and Feldser, David M., primary

Published
2023
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17. Supplementary Figures 1-10. from Systematic In Vivo Inactivation of Chromatin-Regulating Enzymes Identifies Setd2 as a Potent Tumor Suppressor in Lung Adenocarcinoma

Author

Walter, David M., primary, Venancio, Olivia S., primary, Buza, Elizabeth L., primary, Tobias, John W., primary, Deshpande, Charuhas, primary, Gudiel, A. Andrea, primary, Kim-Kiselak, Caroline, primary, Cicchini, Michelle, primary, Yates, Travis J., primary, and Feldser, David M., primary

Published
2023
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19. Supplementary Figure 6 from Tumor-Promoting Desmoplasia Is Disrupted by Depleting FAP-Expressing Stromal Cells

Author

Lo, Albert, primary, Wang, Liang-Chuan S., primary, Scholler, John, primary, Monslow, James, primary, Avery, Diana, primary, Newick, Kheng, primary, O'Brien, Shaun, primary, Evans, Rebecca A., primary, Bajor, David J., primary, Clendenin, Cynthia, primary, Durham, Amy C., primary, Buza, Elizabeth L., primary, Vonderheide, Robert H., primary, June, Carl H., primary, Albelda, Steven M., primary, and Puré, Ellen, primary

Published
2023
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20. Supplementary Figure 8 from Tumor-Promoting Desmoplasia Is Disrupted by Depleting FAP-Expressing Stromal Cells

Author

Lo, Albert, primary, Wang, Liang-Chuan S., primary, Scholler, John, primary, Monslow, James, primary, Avery, Diana, primary, Newick, Kheng, primary, O'Brien, Shaun, primary, Evans, Rebecca A., primary, Bajor, David J., primary, Clendenin, Cynthia, primary, Durham, Amy C., primary, Buza, Elizabeth L., primary, Vonderheide, Robert H., primary, June, Carl H., primary, Albelda, Steven M., primary, and Puré, Ellen, primary

Published
2023
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21. Supplementary Figure 2 from Tumor-Promoting Desmoplasia Is Disrupted by Depleting FAP-Expressing Stromal Cells

Author

Lo, Albert, primary, Wang, Liang-Chuan S., primary, Scholler, John, primary, Monslow, James, primary, Avery, Diana, primary, Newick, Kheng, primary, O'Brien, Shaun, primary, Evans, Rebecca A., primary, Bajor, David J., primary, Clendenin, Cynthia, primary, Durham, Amy C., primary, Buza, Elizabeth L., primary, Vonderheide, Robert H., primary, June, Carl H., primary, Albelda, Steven M., primary, and Puré, Ellen, primary

Published
2023
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22. Supplementary Figure 3 from Tumor-Promoting Desmoplasia Is Disrupted by Depleting FAP-Expressing Stromal Cells

Author

Lo, Albert, primary, Wang, Liang-Chuan S., primary, Scholler, John, primary, Monslow, James, primary, Avery, Diana, primary, Newick, Kheng, primary, O'Brien, Shaun, primary, Evans, Rebecca A., primary, Bajor, David J., primary, Clendenin, Cynthia, primary, Durham, Amy C., primary, Buza, Elizabeth L., primary, Vonderheide, Robert H., primary, June, Carl H., primary, Albelda, Steven M., primary, and Puré, Ellen, primary

Published
2023
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23. Supplementary Figure 1 from Tumor-Promoting Desmoplasia Is Disrupted by Depleting FAP-Expressing Stromal Cells

Author

Lo, Albert, primary, Wang, Liang-Chuan S., primary, Scholler, John, primary, Monslow, James, primary, Avery, Diana, primary, Newick, Kheng, primary, O'Brien, Shaun, primary, Evans, Rebecca A., primary, Bajor, David J., primary, Clendenin, Cynthia, primary, Durham, Amy C., primary, Buza, Elizabeth L., primary, Vonderheide, Robert H., primary, June, Carl H., primary, Albelda, Steven M., primary, and Puré, Ellen, primary

Published
2023
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24. Supplementary Figure 4 from Tumor-Promoting Desmoplasia Is Disrupted by Depleting FAP-Expressing Stromal Cells

Author

Lo, Albert, primary, Wang, Liang-Chuan S., primary, Scholler, John, primary, Monslow, James, primary, Avery, Diana, primary, Newick, Kheng, primary, O'Brien, Shaun, primary, Evans, Rebecca A., primary, Bajor, David J., primary, Clendenin, Cynthia, primary, Durham, Amy C., primary, Buza, Elizabeth L., primary, Vonderheide, Robert H., primary, June, Carl H., primary, Albelda, Steven M., primary, and Puré, Ellen, primary

Published
2023
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25. Supplementary Figure 5 from Tumor-Promoting Desmoplasia Is Disrupted by Depleting FAP-Expressing Stromal Cells

Author

Lo, Albert, primary, Wang, Liang-Chuan S., primary, Scholler, John, primary, Monslow, James, primary, Avery, Diana, primary, Newick, Kheng, primary, O'Brien, Shaun, primary, Evans, Rebecca A., primary, Bajor, David J., primary, Clendenin, Cynthia, primary, Durham, Amy C., primary, Buza, Elizabeth L., primary, Vonderheide, Robert H., primary, June, Carl H., primary, Albelda, Steven M., primary, and Puré, Ellen, primary

Published
2023
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26. Supplementary Table 1 from Tumor-Promoting Desmoplasia Is Disrupted by Depleting FAP-Expressing Stromal Cells

Author

Lo, Albert, primary, Wang, Liang-Chuan S., primary, Scholler, John, primary, Monslow, James, primary, Avery, Diana, primary, Newick, Kheng, primary, O'Brien, Shaun, primary, Evans, Rebecca A., primary, Bajor, David J., primary, Clendenin, Cynthia, primary, Durham, Amy C., primary, Buza, Elizabeth L., primary, Vonderheide, Robert H., primary, June, Carl H., primary, Albelda, Steven M., primary, and Puré, Ellen, primary

Published
2023
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27. Supplementary Materials and Methods from Tumor-Promoting Desmoplasia Is Disrupted by Depleting FAP-Expressing Stromal Cells

Author

Lo, Albert, primary, Wang, Liang-Chuan S., primary, Scholler, John, primary, Monslow, James, primary, Avery, Diana, primary, Newick, Kheng, primary, O'Brien, Shaun, primary, Evans, Rebecca A., primary, Bajor, David J., primary, Clendenin, Cynthia, primary, Durham, Amy C., primary, Buza, Elizabeth L., primary, Vonderheide, Robert H., primary, June, Carl H., primary, Albelda, Steven M., primary, and Puré, Ellen, primary

Published
2023
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28. Data from Tumor-Promoting Desmoplasia Is Disrupted by Depleting FAP-Expressing Stromal Cells

Author

Lo, Albert, primary, Wang, Liang-Chuan S., primary, Scholler, John, primary, Monslow, James, primary, Avery, Diana, primary, Newick, Kheng, primary, O'Brien, Shaun, primary, Evans, Rebecca A., primary, Bajor, David J., primary, Clendenin, Cynthia, primary, Durham, Amy C., primary, Buza, Elizabeth L., primary, Vonderheide, Robert H., primary, June, Carl H., primary, Albelda, Steven M., primary, and Puré, Ellen, primary

Published
2023
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29. Supplementary Figure Legends from Tumor-Promoting Desmoplasia Is Disrupted by Depleting FAP-Expressing Stromal Cells

Author

Lo, Albert, primary, Wang, Liang-Chuan S., primary, Scholler, John, primary, Monslow, James, primary, Avery, Diana, primary, Newick, Kheng, primary, O'Brien, Shaun, primary, Evans, Rebecca A., primary, Bajor, David J., primary, Clendenin, Cynthia, primary, Durham, Amy C., primary, Buza, Elizabeth L., primary, Vonderheide, Robert H., primary, June, Carl H., primary, Albelda, Steven M., primary, and Puré, Ellen, primary

Published
2023
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30. Supplementary Figure 7 from Tumor-Promoting Desmoplasia Is Disrupted by Depleting FAP-Expressing Stromal Cells

Author

Lo, Albert, primary, Wang, Liang-Chuan S., primary, Scholler, John, primary, Monslow, James, primary, Avery, Diana, primary, Newick, Kheng, primary, O'Brien, Shaun, primary, Evans, Rebecca A., primary, Bajor, David J., primary, Clendenin, Cynthia, primary, Durham, Amy C., primary, Buza, Elizabeth L., primary, Vonderheide, Robert H., primary, June, Carl H., primary, Albelda, Steven M., primary, and Puré, Ellen, primary

Published
2023
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31. Immune transgene-dependent myocarditis in macaques after systemic administration of adeno-associated virus expressing human acid alpha-glucosidase

Author

Hordeaux, Juliette, primary, Ramezani, Ali, additional, Tuske, Steve, additional, Mehta, Nickita, additional, Song, Chunjuan, additional, Lynch, Anna, additional, Lupino, Katherine, additional, Chichester, Jessica A., additional, Buza, Elizabeth L., additional, Dyer, Cecilia, additional, Yu, Hongwei, additional, Bell, Peter, additional, Weimer, Jill M., additional, Do, Hung, additional, and Wilson, James M., additional

Published
2023
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33. Efficacy and Safety of a Krabbe Disease Gene Therapy

Author

Hordeaux, Juliette, primary, Jeffrey, Brianne A., additional, Jian, Jinlong, additional, Choudhury, Gourav R., additional, Michalson, Kristofer, additional, Mitchell, Thomas W., additional, Buza, Elizabeth L., additional, Chichester, Jessica, additional, Dyer, Cecilia, additional, Bagel, Jessica, additional, Vite, Charles H., additional, Bradbury, Allison M., additional, and Wilson, James M., additional

Published
2022
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35. Engineering the gut microbiota to treat hyperammonemia

Author

Shen, Ting-Chin David, Albenberg, Lindsey, Bittinger, Kyle, Chehoud, Christel, Chen, Ying-Yu, Judge, Colleen A., Chau, Lillian, Ni, Josephine, Sheng, Michael, Lin, Andrew, Wilkins, Benjamin J., Buza, Elizabeth L., Lewis, James D., Daikhin, Yevgeny, Nissim, Ilana, Yudkoff, Marc, Bushman, Frederic D., and Wu, Gary D.

Published
2015
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37. Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol.

Author

Wang, Lili, Smith, Jeff, Breton, Camilo, Clark, Peter, Zhang, Jia, Ying, Lei, Che, Yan, Lape, Janel, Bell, Peter, Calcedo, Roberto, Buza, Elizabeth L, Saveliev, Alexei, Bartsevich, Victor V, He, Zhenning, White, John, Li, Mingyao, Jantz, Derek, and Wilson, James M

Abstract

Genome editing in non-human primates with a PCSK9-targeted meganuclease lowers cholesterol levels. Clinical translation of in vivo genome editing to treat human genetic diseases requires thorough preclinical studies in relevant animal models to assess safety and efficacy. A promising approach to treat hypercholesterolemia is inactivating the secreted protein PCSK9, an antagonist of the LDL receptor. Here we show that single infusions in six non-human primates of adeno-associated virus vector expressing an engineered meganuclease targeting PCSK9 results in dose-dependent disruption of PCSK9 in liver, as well as a stable reduction in circulating PCSK9 and serum cholesterol. Animals experienced transient, asymptomatic elevations of serum transaminases owing to the formation of T cells against the transgene product. Vector DNA and meganuclease expression declined rapidly, leaving stable populations of genome-edited hepatocytes. A second-generation PCSK9-specific meganuclease showed reduced off-target cleavage. These studies demonstrate efficient, physiologically relevant in vivo editing in non-human primates, and highlight safety considerations for clinical translation. [ABSTRACT FROM AUTHOR]

Published
2022
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38. MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates

Author

Hordeaux, Juliette, primary, Buza, Elizabeth L., additional, Jeffrey, Brianne, additional, Song, Chunjuan, additional, Jahan, Tahsin, additional, Yuan, Yuan, additional, Zhu, Yanqing, additional, Bell, Peter, additional, Li, Mingyao, additional, Chichester, Jessica A., additional, Calcedo, Roberto, additional, and Wilson, James M., additional

Published
2020
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39. Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology

Author

Hordeaux, Juliette, primary, Buza, Elizabeth L., additional, Dyer, Cecilia, additional, Goode, Tamara, additional, Mitchell, Thomas W., additional, Richman, Laura, additional, Denton, Nathan, additional, Hinderer, Christian, additional, Katz, Nathan, additional, Schmid, Ralf, additional, Miller, Rod, additional, Choudhury, Gourav R., additional, Horiuchi, Makoto, additional, Nambiar, Kalyani, additional, Yan, Hanying, additional, Li, Mingyao, additional, and Wilson, James M., additional

Published
2020
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43. Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivogenome editing of PCSK9

Author

Wang, Lili, Breton, Camilo, Warzecha, Claude C., Bell, Peter, Yan, Hanying, He, Zhenning, White, John, Zhu, Yanqing, Li, Mingyao, Buza, Elizabeth L., Jantz, Derek, and Wilson, James M.

Abstract

Gene disruption via programmable, sequence-specific nucleases represents a promising gene therapy strategy in which the reduction of specific protein levels provides a therapeutic benefit. Proprotein convertase subtilisin/kexin type 9 (PCSK9), an antagonist of the low-density lipoprotein (LDL) receptor, is a suitable target for nuclease-mediated gene disruption as an approach to treat hypercholesterolemia. We sought to determine the long-term durability and safety of PCSK9 knockdown in non-human primate (NHP) liver by adeno-associated virus (AAV)-delivered meganuclease following our initial report on the feasibility of this strategy. Six previously treated NHPs and additional NHPs administered AAV-meganuclease in combination with corticosteroid treatment or an alternative AAV serotype were monitored for a period of up to 3 years. The treated NHPs exhibited a sustained reduction in circulating PCSK9 and LDL cholesterol (LDL-c) through the course of the study concomitant with stable gene editing of the PCSK9locus. Low-frequency off-target editing remained stable, and no obvious adverse changes in histopathology of the liver were detected. We demonstrate similar on-target nuclease activity in primary human hepatocytes using a chimeric liver-humanized mouse model. These studies demonstrate that targeted in vivogene disruption exerts a lasting therapeutic effect and provide pivotal data for safety considerations, which support clinical translation.

Published
2021
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46. Systematic In Vivo Inactivation of Chromatin-Regulating Enzymes Identifies Setd2 as a Potent Tumor Suppressor in Lung Adenocarcinoma

Author

Walter, David M., primary, Venancio, Olivia S., additional, Buza, Elizabeth L., additional, Tobias, John W., additional, Deshpande, Charuhas, additional, Gudiel, A. Andrea, additional, Kim-Kiselak, Caroline, additional, Cicchini, Michelle, additional, Yates, Travis J., additional, and Feldser, David M., additional

Published
2017
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48. IL-27 Limits Type 2 Immunopathology Following Parainfluenza Virus Infection

Author

Muallem, Gaia, primary, Wagage, Sagie, additional, Sun, Yan, additional, DeLong, Jonathan H., additional, Valenzuela, Alex, additional, Christian, David A., additional, Harms Pritchard, Gretchen, additional, Fang, Qun, additional, Buza, Elizabeth L., additional, Jain, Deepika, additional, Elloso, M. Merle, additional, López, Carolina B., additional, and Hunter, Christopher A., additional

Published
2017
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