Manufacturing and Characterization of a Recombinant Adeno-Associated Virus Type 8 Reference Standard Material

Abstract : Gene therapy approaches using recombinant adeno-associated virus serotype 2 (rAAV2) and serotype 8 (rAAV8) have achieved significant clinical benefits. The generation of rAAV Reference Standard Materials (RSM) is key to providing points of reference for particle titer, vector genome titer, and infectious titer for gene transfer vectors. Following the example of the rAAV2RSM, here we have generated and characterized a novel RSM based on rAAV serotype 8. The rAAV8RSM was produced using transient transfection, and the purification was based on density gradient ultracentrifugation. The rAAV8RSM was distributed for characterization along with standard assay protocols to 16 laboratories worldwide. Mean titers and 95% confidence intervals
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Submitted on : Monday, August 20, 2018 - 4:24:51 PM
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Eduard Ayuso, Veronique Blouin, Martin Lock, Susan Mcgorray, Xavier Leon, et al.. Manufacturing and Characterization of a Recombinant Adeno-Associated Virus Type 8 Reference Standard Material. Human Gene Therapy, Mary Ann Liebert, 2014, 25 (11), pp.977 - 987. ⟨10.1089/hum.2014.057⟩. ⟨inserm-01847281⟩

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