Search Results - "Spronck, Lisa"
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Therapeutic hFIX Activity Achieved after Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-existing Anti-AAV5 NABs
Published in Molecular therapy. Methods & clinical development (13-09-2019)“…Currently, individuals with pre-existing neutralizing antibodies (NABs) against adeno-associated virus (AAV) above titer of 5 are excluded from systemic…”
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The Biodistribution and Tolerability of rAAV5-miHTT after Bilateral Intra-striatal Delivery to Non-human Primates (S16.006)
Published in Neurology (09-04-2019)“…Abstract only…”
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Effect of Adeno‐associated Viral Vector Mediated APOE Protective Variants Expression on Tau Pathology in a Tau Mouse Model
Published in Alzheimer's & dementia (01-12-2023)“…Background Alzheimer’s disease (AD) is characterized by two main pathological hallmarks in the brain, namely, the presence of amyloid plaques and…”
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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
Published in Blood (06-12-2014)“…Restoration of clotting Factor IX (FIX) expression through gene transfer is a promising option for the treatment of Hemophilia B. We have developed an…”
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L4 Sustained and strong HTT silencing by AAV5-miHTT as therapy for huntington’s disease
Published in Journal of neurology, neurosurgery and psychiatry (01-09-2016)“…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…”
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664. Improving AAV Gene Therapy Safety Analysis: Multiplex LAM-PCR Provide New Insights Into AAV Vector Integration
Published in Molecular therapy (01-05-2015)Get full text
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Non-clinical safety evaluation of AAV5-PBGD in mice and cynomolgus macaques
Published in Toxicology letters (28-08-2013)Get full text
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No longitudinal transmission of AAV5-PBGD vector DNA in mice
Published in Toxicology letters (28-08-2013)Get full text
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