Recombinant adeno-associated virus (rAAV) vectors are a highly promising mechanism for therapeutic gene delivery. However, most industrial cell lines exhibit inefficient gene pack[1]aging, which results in a heterogeneous population of vectors composed of empty, partial[1]ly-loaded, and fully-loaded capsids. Purifying, identifying and quantifying these different species is vital from a production and quality control standpoint. Improperly loaded viral capsids do not produce the desired therapeutic effect, but may still elicit an unintended im[1]mune response. This article and expert panel discussion will focus on a variety of pertinent topics in rAAV process development, with a focus on the benefits of analytical ultracentrif[1]ugation for vector purification and characterization. The article explores challenges of purification and QC within gene therapy with details on DGUC, Iodixanol vs CsCl for AAV, and characterizing AAV with AUC
• Product Interest
Analytical Ultracentrifugation, Ultracentrifugation
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