Adeno-associated virus vectors in gene therapy
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The development of targeted vectors, capable of tissue-specific transduction, remains one of the most important aims of vector modification for gene therapy applications. The gaining popularity of recombinant vectors based on adeno-associated viruses (rAAV) in gene therapy can be attributed to their lack of pathogenicity, added safety due to their replication defectiveness, relatively low immunogenicity and their ability to mediate long-term expression in a variety of tissues. The major shortcoming of these vectors is their small packaging capacity. AAV vectors have already broad utility in the therapy of many diseases, including neurological disorders and various types of cancer. Moreover, they can also serve as transfer vehicles for DNA vaccines.
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Kinga Kamieniarz, Zaklad Wirusologii Molekularnej, Instytut Biologii Molekularnej i Biotechnologii, Uniwersytet im. Adama Mickiewicza, ul. Miedzychodzka 5, 60-371 Poznan, Poland