Therapuetic effector functions of AAV expressed transgenes

腺相关病毒（AAV）载体具有替代常规抗逆转录病毒疗法的潜力，或 甚至可以预防最初的HIV-1感染。AAV载体的潜力来自两种性质： 特殊的安全性，以及它们多年来维持非常高水平的转基因表达的能力。 自身互补的AAV（scAAV）载体可以维持100-200 pg/ml的蛋白抑制剂的表达，以用于表达蛋白抑制剂。 两年多相比之下，来自常规单链（ssAAV）载体的转基因表达不受限制。 是原来的十倍多然而，scAAV转基因的大小必须是ssAAV转基因的一半。 这种限制排除了全长抗体的表达，而是需要使用非天然抗体样多肽。 分子如单链免疫粘附素（scFv-Fc）。此外，scAAV载体的大小限制 防止包含其它有用的分子，例如伊加必需的连接（J）链 多聚化，以及可用于各种

Adeno-associated virus (AAV) vectors have the potential to replace conventional anti-retroviral therapies, or even protect against an initial HIV-1 infection. The potential of AAV vectors arises from two properties: their exceptional safety profile, and their ability to sustain very high levels of transgene expression for years. A self-complementary AAV (scAAV) vector can sustain expression of 100-200 pg/ml of protein inhibitors for more than two years. In contrast, transgene expression from a conventional, single-stranded (ssAAV) vector is more than ten-fold lower. However, scAAV transgenes are necessarily half the size of ssAAV transgenes. This limit precludes expression of full-length antibodies, and instead requires use of non-native antibody-like molecules such as single-chain immunadhesins (scFv-Fc). Moreover, the size limitation of scAAV vectors prevents inclusion of other useful molecules, for example the joining (J) chains essential for IgA multimerization, and proteins and regulatory regions useful in various