Optimization of methods for the production of AAV vectors
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One of the conditions of effective gene therapy is the choice of a proper gene carrier that will efficiently deliver the genetic material to the damaged tissue without causing deleterious side-effects. Adeno-associated viral vectors (AAV) have emerged as attractive tools for gene therapy, because of their broad tissue tropism, long-term transgene expression, and lack of human pathology. Nevertheless, difficulty in preparing and purifying this viral vector in large quantities remains a major obstacle for evaluating AAV vectors in clinical trials. In this article, we compare different methods for AAV production in order to optimize the conditions of AAV preparation to the scale and purity required for clinical and potential commercial applications.
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Jozef Dulak, Zaklad Biotechnologii Medycznej, Wydzial Biochemii, Biofizyki i Biotechnologii, Uniwersytet Jagiellonski, ul. Gronostajowa 7, 31-271 Krakow, Poland