Recombinant adeno-associated viral vectors (rAAV) are probably the most powerful tools for in vivo gene delivery. Encouraging preclinical data have been followed by successful gene therapy clinical trials including Leber's congenital amaurosis type 2 (refs. 1–3), hemophilia B,4,5 and recently choroideremia.6 These results together with the market authorization of Glybera, an AAV-based product for the treatment of lipoprotein lipase deficiency,7,8 has prompted skeptical investors and biotechnology and pharmaceuticals companies to move into this field.
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Eduard Ayuso (2016) studied this question.
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