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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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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Journal Article
Defining phenotype reversibility in lysosomal disease: Leveraging a COIN model in mucopolysaccharidosis type VI (MPS VI)
Hwang-Wong, Elizabeth, Amar, Gabrielle, Das, Nanditha, Zhang, Xiaoli, Rothman, Nyanza, Calafati, Philip, Aaron, Nina, Gale, Kirsten, Fante, Massimo, Lees-Shepard, John, Nannuru, Kalyan, Baik, Andrew, 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
B형 혈우병 치료를 위한 인자 IX를 발현하기 위한 조성물 및 방법
KATAKOWSKI JOSEPH, CYGNAR KATHERINE, KYRATSOUS CHRISTOS, PEFANIS EVANGELOS, CALAFATI PHILIP, SABIN LEAH, SAMAI POULAMI, BAIK ANDREW, MOLLER TANK SVEN
Year of Publication 28.06.2024
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Year of Publication 28.06.2024
Patent
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
CRISPR-MEDIATED TRANSGENE INSERTION IN NEONATAL CELLS
SAMAI, Poulami, CALAFATI, Philip, CYGNAR, Katherine, SABIN, Leah, BAIK, Andrew
Year of Publication 10.08.2023
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Year of Publication 10.08.2023
Patent
ANTI-TFR:GAA AND ANTI-CD63:GAA INSERTIONS FOR TREATMENT OF POMPE DISEASE
CALAFATI, Philip, KEATING, Nicole, FORGET, Anthony, PRAGGASTIS, Maria, BABB, Robert, BAIK, Andrew, SAMAI, Poulami, GAO, Min, CYGNAR, Katherine, AIMÉ-WILSON, Pascaline, DUGAN, John, SABIN, Leah, PEFANIS, Evangelos
Year of Publication 11.07.2024
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Year of Publication 11.07.2024
Patent
ANTI-TFR:GAA AND ANTI-CD63:GAA INSERTION FOR TREATMENT OF POMPE DISEASE
Forget, Anthony, Cygnar, Katherine, Calafati, Philip, Pefanis, Evangelos, Sabin, Leah, Keating, Nicole, Praggastis, Maria, Aimé-Wilson, Pascaline, Gao, Min, Samai, Poulami, Dugan, John, Baik, Andrew, Babb, Robert
Year of Publication 26.10.2023
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Year of Publication 26.10.2023
Patent
ANTI-TFR:GAA AND ANTI-CD63:GAA INSERTIONS FOR TREATMENT OF POMPE DISEASE
CALAFATI, Philip, KEATING, Nicole, FORGET, Anthony, PRAGGASTIS, Maria, BABB, Robert, BAIK, Andrew, SAMAI, Poulami, GAO, Min, CYGNAR, Katherine, AIMÉ-WILSON, Pascaline, DUGAN, John, SABIN, Leah, PEFANIS, Evangelos
Year of Publication 28.09.2023
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Year of Publication 28.09.2023
Patent
ANTI-TFR:GAA AND ANTI-CD63:GAA INSERTION FOR TREATMENT OF POMPE DISEASE
CALAFATI, Philip, KEATING, Nicole, FORGET, Anthony, PRAGGASTIS, Maria, BABB, Robert, BAIK, Andrew, SAMAI, Poulami, GAO, Min, CYGNAR, Katherine, AIMÉ-WILSON, Pascaline, DUGAN, John, SABIN, Leah, PEFANIS, Evangelos
Year of Publication 10.08.2023
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Year of Publication 10.08.2023
Patent
COMPOSITIONS AND METHODS FOR EXPRESSING FACTOR IX FOR HEMOPHILIA B THERAPY
SAMAI Poulami, BAIK Andrew, SABIN Leah, CALAFATI Philip, PEFANIS Evangelos, KATAKOWSKI Joseph, KYRATSOUS Christos, CYGNAR Katherine, MOLLER-TANK Sven
Year of Publication 16.05.2024
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Year of Publication 16.05.2024
Patent