Search Results - "Bouwman, Taren"
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1
The longitudinal kinetics of AAV5 vector integration profiles and evaluation of clonal expansion in mice
Published in Molecular therapy. Methods & clinical development (12-09-2024)“…Adeno-associated virus (AAV)-based vectors are used clinically for gene transfer and persist as extrachromosomal episomes. A small fraction of vector genomes…”
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2
Young mice administered adult doses of AAV5-hFVIII-SQ achieve therapeutic factor VIII expression into adulthood
Published in Molecular therapy. Methods & clinical development (08-09-2022)“…Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) gene transfer provided reduced bleeding for adult clinical trial participants with severe hemophilia A. However,…”
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3
Vector genome loss and epigenetic modifications mediate decline in transgene expression of AAV5 vectors produced in mammalian and insect cells
Published in Molecular therapy (07-12-2022)“…Recombinant adeno-associated virus (rAAV) vectors are often produced in HEK293 or Spodoptera frugiperda (Sf)-based cell lines. We compared expression profiles…”
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Journal Article