Gene Therapy in Mice: Subretinal vs. Intravitreal AAV Vector Delivery
Author Information
Author(s): Li Wensheng, Kong Fansheng, Li Xia, Dai Xufeng, Liu Xiaoqiang, Zheng Qinxiang, Wu Ronghan, Zhou Xiangtian, Lü Fan, Chang Bo, Li Qiuhong, Hauswirth William W., Qu Jia, Pang Ji-jing
Primary Institution: Wenzhou Medical College
Hypothesis
Does a previous intravitreal injection affect the efficiency of subretinal AAV vector transduction in the partner eye?
Conclusion
An initial intravitreal injection of AAV vectors does not influence gene expression or therapeutic effects in the other eye when vectors are administered subretinally.
Supporting Evidence
- Subretinal injections resulted in strong gene expression in both C57BL/6J and rd12 mice.
- ERG restoration was statistically similar in treated rd12 eyes regardless of prior intravitreal injection.
- RPE65 expression was significantly higher in subretinally injected eyes compared to intravitreal ones.
Takeaway
This study shows that giving a shot in one eye doesn't hurt the treatment in the other eye when using a special kind of gene therapy.
Methodology
Mice received intravitreal injections followed by subretinal injections, and their retinal function was evaluated using electroretinograms.
Limitations
Results may not directly translate to humans due to differences in immune response and retinal structure.
Participant Demographics
C57BL/6J and rd12 mice, aged four to eight weeks.
Statistical Information
P-Value
p<0.001
Statistical Significance
p<0.001
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