Reduction of TMEM97 increases NPC1 protein levels and restores cholesterol trafficking in Niemann-pick type C1 disease cells
Ebrahimi-Fakhari, Darius, Wahlster, Lara, Bartz, Fabian, Werenbeck-Ueding, Jennifer, Praggastis, Maria, Zhang, Jessie, Joggerst-Thomalla, Brigitte, Theiss, Susanne, Grimm, Dirk, Ory, Daniel S, Runz, Heiko
Published in Human molecular genetics (15.08.2016)
Published in Human molecular genetics (15.08.2016)
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Journal Article
A murine Niemann-Pick C1 I1061T knock-in model recapitulates the pathological features of the most prevalent human disease allele
Praggastis, Maria, Tortelli, Brett, Zhang, Jessie, Fujiwara, Hideji, Sidhu, Rohini, Chacko, Anita, Chen, Zhouji, Chung, Chan, Lieberman, Andrew P, Sikora, Jakub, Davidson, Cristin, Walkley, Steven U, Pipalia, Nina H, Maxfield, Frederick R, Schaffer, Jean E, Ory, Daniel S
Published in The Journal of neuroscience (27.05.2015)
Published in The Journal of neuroscience (27.05.2015)
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Journal Article
Delivering therapeutic lysosomal enzyme to the CNS: A rapid, large-scale screen identifies optimal characteristics of BBB-crossing TFRC antibodies
Praggastis, Maria, Gale, Kirsten, Baik, Andrew, Aaron, Nina, Rothman, Nyanza, Zhang, Xiaoli, Giovannone, Nicholas, Hwang-Wong, Elizabeth, Cygnar, Katherine D.
Published in Molecular genetics and metabolism (01.02.2023)
Published in Molecular genetics and metabolism (01.02.2023)
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Journal Article
Delivering therapeutic lysosomal enzyme to the CNS: A rapid, large-scale in vivo screen identifies optimal characteristics of BBB-crossing TFRC antibodies
Praggastis, Maria, Gale, Kirsten, Baik, Andrew, Aaron, Nina, Rothman, Nyanza, Zhang, Xiaoli, Giovannone, Nicholas, Hwang-Wong, Elizabeth, Cygnar, Katherine D.
Published in Molecular genetics and metabolism (01.02.2023)
Published in Molecular genetics and metabolism (01.02.2023)
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Journal Article
BBB-targeted GAA delivered as gene therapy treats CNS and muscle in Pompe disease model mice
Praggastis, Maria, Gale, Kirsten, Baik, Andrew, Calafati, Philip, Aaron, Nina, Rothman, Nyanza, Zhang, Xiaoli, Giovannone, Nicholas, Hwang, Elizabeth, Economides, Aris, Cygnar, Katherine
Published in Molecular genetics and metabolism (01.02.2022)
Published in Molecular genetics and metabolism (01.02.2022)
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Journal Article
Cell type-selective targeted delivery of a recombinant lysosomal enzyme for enzyme therapies
Baik, Andrew D., Calafati, Philip, Zhang, Xiaoli, Aaron, Nina A., Mehra, Antonia, Moller-Tank, Sven, Miloscio, Lawrence, Praggastis, Maria, Giovannone, Nicholas, Pan, Cheryl, Tang, Yajun, Bridges, Susannah, Mujica, Alejo, Barbounis, Peter, Yanolatos, Jean, Gale, Nicholas, Li, Ning, Kyratsous, Christos A., Schoenherr, Christopher J., Murphy, Andrew J., Economides, Aris N., Cygnar, Katherine D.
Published in Molecular therapy (01.12.2021)
Published in Molecular therapy (01.12.2021)
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폼페병의 치료를 위한 항-TfR:GAA 및 항-CD63:GAA 삽입
PRAGGASTIS MARIA, KEATING NICOLE, CYGNAR KATHERINE, GAO MIN, AIME WILSON PASCALINE, FORGET ANTHONY, DUGAN JOHN, PEFANIS EVANGELOS, BABB ROBERT, CALAFATI PHILIP, SABIN LEAH, SAMAI POULAMI, BAIK ANDREW
Year of Publication 11.09.2024
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Year of Publication 11.09.2024
Patent
A Murine Niemann-Pick C1 I1061T Knock-In Model Recapitulates the Pathological Features of the Most Prevalent Human Disease Allele
Praggastis, Maria, Tortelli, Brett, Zhang, Jessie, Fujiwara, Hideji, Sidhu, Rohini, Chacko, Anita, Chen, Zhouji, Chung, Chan, Lieberman, Andrew P, Sikora, Jakub
Published in The Journal of neuroscience (27.05.2015)
Published in The Journal of neuroscience (27.05.2015)
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Journal Article
NON-HUMAN ANIMALS COMPRISING A MODIFIED TRANSFERRIN RECEPTOR LOCUS
CYGNAR, Katherine, PRAGGASTIS, Maria, FANG, Qing, TANG, Yajun, ZHONG, Jun, MURPHY, Andrew J, MUJICA, Alexander O
Year of Publication 04.04.2024
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Year of Publication 04.04.2024
Patent