Vectors derived from adeno-associated viruses (AAV) represent a promising tool for retinal gene transfer in pre-clinical and clinical settings. AAV vectors efficiently transduce dividing and non-dividing cells, escape cellular immunity and result in long-non-term transduction. In addition, they may be targeted to specific retinal cell types by taking advantage of surface proteins from various AAV serotypes thus limiting transfer of therapeutic genes to those cells requiring correction. This review will provide an overview of the properties of AAV vectors followed by a detailed report of their use in retinal gene transfer for mendelian and non-mendelian disorders.

中文翻译：

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用于视网膜基因转移的腺相关病毒载体。

来自腺相关病毒 (AAV) 的载体代表了在临床前和临床环境中进行视网膜基因转移的有前途的工具。AAV 载体有效地转导分裂和非分裂细胞，逃避细胞免疫并导致长期非长期转导。此外，它们可以通过利用来自各种 AAV 血清型的表面蛋白来靶向特定的视网膜细胞类型，从而限制治疗基因向那些需要校正的细胞的转移。这篇综述将概述 AAV 载体的特性，然后详细报告它们在孟德尔和非孟德尔疾病的视网膜基因转移中的应用。