The eye is the most important organ of the human senses. Compared with other organs, the eye is small, relatively closed and away from other tissues. Eye diseases are complex, and the rise of gene therapy offers hope for the treatment of inherited eye diseases (retinitis pigmentosa, Leber congenital amaurosis, etc.), even pathological eye diseases (retinopathy, etc.), and other eye diseases (optic nerve damage).
Adeno-associated virus (AAV) is a ideal gene transfer vector tool in gene therapy of eye diseases due to its advantages such as good safety, wide host range, low immunogenicity. At present, there are at least 9 serotypes of AAV used for gene transfer in the eyes, and each serotype of AAV has different infection efficiency for different tissues and cells. To more efficiently and specifically infect the retina and retinal Mueller cells, the scientists developed retinal specific serotype AAV-ANC80L65 and retinal mueller cell specific serotype AAV-SHH10 by analyzing capsid proteins of existing serotypes.
1. rAAVs serotype anc80L65 vectors highly infected with retina in vivo.
Reference：Synthetic adeno-associated viral vector efficiently targets mouse and non-human primate retina in vivo.
AAV2, AAV8, Anc80L27, Anc80L65 and Anc80L121 were injected into male C57Bl/6 mice (6-8 weeks old) at equal doses. The expression of the EGFP fluorescent protein was monitored through  fundus imaging of the retina after 4 weeks post-injection. Results show Muller glia transduction for all serotypes, except AAV2, and for Anc80 variants a number  of additional neuronal retinal cell types were clearly GFP positive.