Abstract
Purpose :
With an increasing number of identified causative genes, the widespread use of gene therapy becomes feasible. Once a target gene is selected, it is important to have a cell delivery method that is both specific and efficient. The need for cell type specificity and high efficiency is particularly important for treating retinal degenerations, which may result from defects in the pigmented epithelium, in rods, or in cones. Viruses are efficient gene delivery vehicles for the nervous system, but they often suffer from non-specific infection. To address specificity and efficiency in targeting retinal photoreceptors, we have screened various adeno-associated virus (AAV) serotypes for infection patterns in the mouse retina following subretinal injection.
Methods :
We injected AAVs (2×108μg/μL) to subretinal layer of 1M mouse retina. Dissected retina 7days later and performed immunohistochemistry. All analyses were performed with an LSM700 confocal microscopy (Carl Zeiss, Oberkochen, Germany).
Results :
We have detected AAV2/6 infected dominantly in cone photoreceptors. There were no preference between cone photoreceptor serotypes.
Conclusions :
Our study suggest AAV2/6 would be a useful tool for delivering genes to cone photoreceptors, in combination with cone specific promoters that already reported, for the purpose of restoring genes involving in retinal degeneration.
This abstract was presented at the 2019 ARVO Annual Meeting, held in Vancouver, Canada, April 28 - May 2, 2019.