Výsledky vyhledávání - "Lisa Spronck"

Therapeutic hFIX Activity Achieved after Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-existing Anti-AAV5 NABs

Autor: Christian F. Meyer, Bart A. Nijmeijer, Valerie Ferreira, Martin de Haan, Margit H. Lampen, Anna Majowicz, Marco Tangelder, Lisa Spronck, Harald Petry, Sander J. H. van Deventer

Publikováno v: Molecular Therapy. Methods & Clinical Development

Currently, individuals with pre-existing neutralizing antibodies (NABs) against adeno-associated virus (AAV) above titer of 5 are excluded from systemic AAV-based clinical trials. In this study we explored the impact of pre-existing anti-AAV5 NABs on

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::01b473ab29a9d676eabe47a96eb30bf7
https://doi.org/10.1016/j.omtm.2019.05.009

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664. Improving AAV Gene Therapy Safety Analysis: Multiplex LAM-PCR Provide New Insights Into AAV Vector Integration

Autor: Esperanza Lopez-Franco, Manfred G. Schmidt, Christine Käppel, Christof von Kalle, Irene Gil-Farina, Harald Petry, Gloria González-Aseguinolaza, Jesús Prieto, Lisa Spronck, Stefan Wilkening, Maria Astrid Paneda

Publikováno v: Molecular Therapy. 23

Nowadays, recombinant adeno-associated viruses (rAAV) constitute one of the most successful vectors for human gene therapy. These viral vectors allow a long-term transgene expression mainly due to their ability to form stable concatemeric DNA structu

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::208416bafb50277916b8257504bf4952

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L4 Sustained and strong HTT silencing by AAV5-miHTT as therapy for huntington’s disease

Autor: Cynthia Brouwers, Pavlina Konstantinova, Nicole Déglon, Martin de Haan, Charles Richard, Krystof S. Bankiewicz, Bas Blits, Melvin M. Evers, Lisa Spronck, Harald Petry, Jana Miniarikova, Jan Motlik, Amber L. Southwell

Publikováno v: Journal of Neurology, Neurosurgery & Psychiatry. 87:A91.2-A91

Huntington’s disease (HD) is an autosomal dominant neurodegenerative disorder caused by a CAG trinucleotide repeat expansion in the first exon of the HTT gene. This CAG repeat expansion results in an expanded polyglutamine repeat in the huntingtin

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::6554e9b8379e843a231205381e0bf0c9
https://doi.org/10.1136/jnnp-2016-314597.259

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Safety and Efficacy of an Adeno Associated Virus-Based Vector Carrying the Human Clotting Factor IX Gene (AAV5-hFIX) As Studied in Non-Human Primates

Autor: Florence Salmon, Harald Petry, Lisa Spronck, Jørgen Holm Petersen, Corina Van Der Kruijssen, Bart A. Nijmeijer

Publikováno v: Blood. 124:4800-4800

Restoration of clotting Factor IX (FIX) expression through gene transfer is a promising option for the treatment of Hemophilia B. We have developed an Adeno-Associated Virus serotype 5-based vector containing the human coagulation factor IX gene (AAV

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::56cdc25614741eb67a4f2df3d3e5a638
https://doi.org/10.1182/blood.v124.21.4800.4800

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