Systemic delivery of a glucosylceramide synthase inhibitor reduces CNS substrates and increases lifespan in a mouse model of type 2 Gaucher disease
Cabrera-Salazar, Mario A, Deriso, Matthew, Bercury, Scott D, Li, Lingyun, Lydon, John T, Weber, William, Pande, Nilesh, Cromwell, Mandy A, Copeland, Diane, Leonard, John, Cheng, Seng H, Scheule, Ronald K
Published in PloS one (17.08.2012)
Published in PloS one (17.08.2012)
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Journal Article
Iminosugar-based inhibitors of glucosylceramide synthase increase brain glycosphingolipids and survival in a mouse model of Sandhoff disease
Ashe, Karen M, Bangari, Dinesh, Li, Lingyun, Cabrera-Salazar, Mario A, Bercury, Scott D, Nietupski, Jennifer B, Cooper, Christopher G F, Aerts, Johannes M F G, Lee, Edward R, Copeland, Diane P, Cheng, Seng H, Scheule, Ronald K, Marshall, John
Published in PloS one (29.06.2011)
Published in PloS one (29.06.2011)
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Journal Article
Merits of Combination Cortical, Subcortical, and Cerebellar Injections for the Treatment of Niemann-Pick Disease Type A
Bu, Jie, Ashe, Karen M, Bringas, John, Marshall, John, Dodge, James C, Cabrera-Salazar, Mario A, Forsayeth, John, Schuchman, Edward H, Bankiewicz, Krystof S, Cheng, Seng H, Shihabuddin, Lamya S., Passini, Marco A
Published in Molecular therapy (01.10.2012)
Published in Molecular therapy (01.10.2012)
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Journal Article
Timing of Therapeutic Intervention Determines Functional and Survival Outcomes in a Mouse Model of Late Infantile Batten Disease
Cabrera-Salazar, Mario A, Roskelley, Eric M, Bu, Jie, Hodges, Bradley L, Yew, Nelson, Dodge, James C, Shihabuddin, Lamya S, Sohar, Istvan, Sleat, David E, Scheule, Ronald K, Davidson, Beverly L, Cheng, Seng H, Lobel, Peter, Passini, Marco A
Published in Molecular therapy (01.10.2007)
Published in Molecular therapy (01.10.2007)
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Journal Article
893. Second Generation Gene Therapy Vector for Classical Late Infantile Neuronal Ceroid Lipofuscinosis
Cabrera-Salazar, Mario A., Hodges, Bradley L., Yew, Nelson S., Bu, Jie, Fidler, Jonathan A., Roskelley, Eric M., Scheule, Ronald K., Sleat, David E., Lobel, Peter, Cheng, Seng H., Passini, Marco A.
Published in Molecular therapy (01.05.2006)
Published in Molecular therapy (01.05.2006)
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Journal Article
Gene therapy for the lysosomal storage disorders
Cabrera-Salazar, Mario A, Novelli, Enrico, Barranger, John A
Published in Current opinion in molecular therapeutics (01.08.2002)
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Published in Current opinion in molecular therapeutics (01.08.2002)
Journal Article
Merits of Combination Cortical, Subcortical, and Cerebellar Injections for the Treatment of Niemann-Pick Disease Type A
Bu, Jie, Ashe, Karen M, Bringas, John, Marshall, John, Dodge, James C, Cabrera-Salazar, Mario A, sayeth, John, Schuchman, Edward H, Bankiewicz, Krystof S, Cheng, Seng H, Shihabuddin, Lamya S, Passini, Marco A
Published in Molecular therapy (01.10.2012)
Published in Molecular therapy (01.10.2012)
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Journal Article
Glucosylceramide synthase inhibitors
Good, Andrew, Makino, Elina, Cheng, Seng H, Husson, Herve, Leonard, John, Celatka, Cassandra, Marshall, John, Beskrovnaya, Oxana, Scheule, Ronald K, Xiang, Yibin, Skerlj, Renato, Jancsics, Katherine, Cabrera-Salazar, Mario A, Natoli, Thomas, Bourque, Elyse, Zhao, Zhong, Hirth, Bradford, Metz, Markus
Year of Publication 13.08.2024
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Year of Publication 13.08.2024
Patent
GLUCOSYLCERAMIDE SYNTHASE INHIBITORS
SKERLJ, RENATO, MARSHALL, JOHN, CHENG, SENG H, LEONARD, JOHN, NATOLI, THOMAS, BOURQUE, ELYSE, XIANG, YIBIN, HIRTH, BRADFORD, JANCSICS, KATHERINE, HUSSON, HERVE, BESKROVNAYA, OXANA, SCHEULE, RONALD K, METZ, MARKUS, CABRERA-SALAZAR, MARIO A, CELATKA, CASSANDRA, ZHAO, ZHONG, MAKINO, ELINA, GOOD, ANDREW
Year of Publication 17.10.2023
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Year of Publication 17.10.2023
Patent
GLUCOSYLCERAMIDE SYNTHASE INHIBITORS
SKERLJ, RENATO, MARSHALL, JOHN, CHENG, SENG H, LEONARD, JOHN, NATOLI, THOMAS, BOURQUE, ELYSE, XIANG, YIBIN, HIRTH, BRADFORD, JANCSICS, KATHERINE, HUSSON, HERVE, BESKROVNAYA, OXANA, SCHEULE, RONALD K, METZ, MARKUS, CABRERA-SALAZAR, MARIO A, CELATKA, CASSANDRA, ZHAO, ZHONG, MAKINO, ELINA, GOOD, ANDREW
Year of Publication 03.10.2022
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Year of Publication 03.10.2022
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GLUCOSYLCERAMIDE SYNTHASE INHIBITORS
SKERLJ, RENATO, MARSHALL, JOHN, CABRERA-SALAZAR, MARIO, A, LEONARD, JOHN, NATOLI, THOMAS, BOURQUE, ELYSE, XIANG, YIBIN, HIRTH, BRADFORD, JANCSICS, KATHERINE, HUSSON, HERVE, BESKROVNAYA, OXANA, CHENG, SENG, H, SCHEULE, RONALD, K, METZ, MARKUS, CELATKA, CASSANDRA, ZHAO, ZHONG, MAKINO, ELINA, GOOD, ANDREW
Year of Publication 31.05.2022
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Year of Publication 31.05.2022
Patent
GLUCOSYLCERAMIDE SYNTHASE INHIBITORS
Good, Andrew, Makino, Elina, Cheng, Seng H, Husson, Herve, Leonard, John, Celatka, Cassandra, Marshall, John, Beskrovnaya, Oxana, Scheule, Ronald K, Xiang, Yibin, Skerlj, Renato, Jancsics, Katherine, Cabrera-Salazar, Mario A, Natoli, Thomas, Bourque, Elyse, Zhao, Zhong, Hirth, Bradford, Metz, Markus
Year of Publication 13.05.2022
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Year of Publication 13.05.2022
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GLUCOSYLCERAMIDSYNTHASE-INHIBITORER
BESKROVNAYA, Oxana, CELATKA, Cassandra, LEONARD, John, SKERLJ, Renato, BOURQUE, Elyse, MAKINO, Elina, METZ, Markus, Xiang, Yibin, NATOLI, Thomas, GOOD, Andrew, CABRERA-SALAZAR, Mario, A, HIRTH, Bradford, CHENG, Seng, H, SCHEULE, Ronald, K, HUSSON, Herve, MARSHALL, John, ZHAO, Zhong, JANCSICS, Katherine
Year of Publication 09.05.2022
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Year of Publication 09.05.2022
Patent
GLUCOSYLCERAMIDE SYNTHASE INHIBITORS
CELATKA Cassandra, MARSHALL John, LEONARD John, CHENG Seng H, SCHEULE Ronald K, NATOLI Thomas, HUSSON Herve, JANCSICS Katherine, CABRERA-SALAZAR Mario A, ZHAO Zhong, BESKROVNAYA Oxana, BOURQUE Elyse, XIANG Yibin, SKERLJ Renato, HIRTH Bradford, GOOD Andrew, METZ Markus, MAKINO Elina
Year of Publication 29.04.2022
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Year of Publication 29.04.2022
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GLUCOSYLCERAMIDE SYNTHASE INHIBITORS
BESKROVNAYA, Oxana, CELATKA, Cassandra, XIANG, Yibin, LEONARD, John, BOURQUE, Elyse, CHENG, Seng H, SKERLJ, Renato, CABRERA-SALAZAR, Mario A, MAKINO, Elina, METZ, Markus, NATOLI, Thomas, GOOD, Andrew, HIRTH, Bradford, SCHEULE, Ronald K, HUSSON, Herve, MARSHALL, John, ZHAO, Zhong, JANCSICS, Katherine
Year of Publication 10.03.2022
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Year of Publication 10.03.2022
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GLUCOSYLCERAMIDE SYNTHASE INHIBITORS
BESKROVNAYA, Oxana, CELATKA, Cassandra, XIANG, Yibin, LEONARD, John, BOURQUE, Elyse, SKERLJ, Renato, MAKINO, Elina, METZ, Markus, NATOLI, Thomas, GOOD, Andrew, CABRERA-SALAZAR, Mario, A, HIRTH, Bradford, CHENG, Seng, H, SCHEULE, Ronald, K, HUSSON, Herve, MARSHALL, John, ZHAO, Zhong, JANCSICS, Katherine
Year of Publication 23.02.2022
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Year of Publication 23.02.2022
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GLUCOSYLCERAMIDE SYNTHASE INHIBITORS
SKERLJ, RENATO, MARSHALL, JOHN, CABRERA-SALAZAR, MARIO, A, LEONARD, JOHN, NATOLI, THOMAS, BOURQUE, ELYSE, XIANG, YIBIN, HIRTH, BRADFORD, JANCSICS, KATHERINE, HUSSON, HERVE, BESKROVNAYA, OXANA, CHENG, SENG, H, SCHEULE, RONALD, K, METZ, MARKUS, CELATKA, CASSANDRA, ZHAO, ZHONG, MAKINO, ELINA, GOOD, ANDREW
Year of Publication 23.08.2021
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Year of Publication 23.08.2021
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