Abstract
Adeno-associated viruses (AAV) are widely used as a recombinant vectors in gene therapy.
AAVs are non-pathogenic. They present reduced cytotoxicity and can transduce both dividing and
non-dividing cells. The existence of different serotypes provides flexibility for targeting different tissues
and organs. Its therapeutic success was already shown by the approval of three products by the
European and American regulatory agencies. To satisfy the high dosage, safety, and reproducibility
required in each clinical trial, production platforms based on stable mammalian cell lines have been
proposed as the best strategy. However, the methodologies employed must be adapted to each cell
line, which often results in distinct productivities. In this article, we review the published and commercially
available mammalian stable cell lines, discussing the key factors that impact viral production
yields, such as integration sites and copy numbers.
Keywords:
AAV, gene therapy, stable cell line, viral vector production, manufacturing, AAV yields.
Graphical Abstract
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,
Joana Diogo and
Ana Sofia Coroadinha*
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