Recombinant adeno-associated virus (rAAV) has been widely used for gene delivery in animal models and successfully applied in clinical trials for treating inherited retinal disease. Although subretinal delivery of AAVs can effectively transduce photoreceptors and/or retinal pigmental epithelium (RPE), cells most affected by inherited retinal diseases, the procedure is invasive and complicated, and only delivers the gene to a limited retinal area. AAVs can also be delivered intravitreally to the retina, a much less invasive nonsurgical procedure. However, intravitreal administration of non-modified AAV serotypes tends to transduce only ganglion cells and inner nuclear layer cells. To date, most non-modified AAV serotypes that have been identified are incapable of efficiently transducing photoreceptors and/or RPE when delivered intravitreally. In this study, we investigate the retinal tropism of AAVrh10 vector administered by intravitreal injection to mouse, rat, and rabbit eyes. Our results demonstrate that AAVrh10 is capable of transducing not only inner retinal cells, but also outer retinal cells in all three species, though the transduction efficiency in rabbit was low. In addition, AAVrh10 preferentially transduced outer retinal cells in mouse models of retinal disease. Therefore, AAVrh10 vector could be a useful candidate to intravitreally deliver genes to photoreceptor and RPE cells.

重组腺相关病毒（rAAV）已广泛用于动物模型中的基因递送，并成功应用于治疗遗传性视网膜疾病的临床试验。虽然 AAV 的视网膜下递送可以有效地转导光感受器和/或视网膜色素上皮 (RPE)（受遗传性视网膜疾病影响最大的细胞），但该过程具有侵入性且复杂，并且只能将基因递送到有限的视网膜区域。 AAV 还可以通过玻璃体内输送到视网膜，这是一种侵入性小得多的非手术程序。然而，玻璃体内施用未经修饰的 AAV 血清型往往仅转导神经节细胞和内核层细胞。迄今为止，大多数已鉴定的未修饰 AAV 血清型在玻璃体内递送时无法有效转导光感受器和/或 RPE。在这项研究中，我们研究了通过玻璃体内注射给小鼠、大鼠和兔眼施用的 A​​AVrh10 载体的视网膜向性。我们的结果表明，AAVrh10 不仅能够转导所有三个物种的内视网膜细胞，而且能够转导外视网膜细胞，尽管在兔子中的转导效率较低。此外，AAVrh10 在视网膜疾病小鼠模型中优先转导外层视网膜细胞。因此，AAVrh10 载体可能是玻璃体内将基因传递到感光细胞和 RPE 细胞的有用候选者。