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Xiaofang Liang, Fangtian Dong, Huajin Li, Qi Zhou, Lizhu Yang, Jijing Pang, Ruifang Sui; AAV5-mediated gene therapy rescue cone function in a mouse model of achromatopsia. Invest. Ophthalmol. Vis. Sci. 2014;55(13):3319.
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© ARVO (1962-2015); The Authors (2016-present)
To clarify whether AAV5 vector-mediated gene therapy could restore cone function in the cnga3cpfl5 mouse model of achromatopsia.
AAV5-IRBP/ GNAT2-hCNGA3 were injected subretinally into one eye of mice at postnatal day 21. The mice that had more than 80% retinal detachment and minimal complications after surgery were kept for follow-up experiments. Contralateral eyes were uninjected as controls. Electroretinographic were recorded to detect cone response at 170 days after treating. Then eyes were enucleated after sacrifice and prepared for cryosections. Opsins expression of retina were conducted with immunofluorescence using S-opsins and M-opsins as primary antibodies.
Compared with the untreated eyes, AAV5 mediated gene therapy could rescue cone response evidently. Both M- and S-opsins were expressed in outer segment of the retina. The curative effect of gene therapy for cnga3cpfl5 lasted for at least 170 days after injection.
In this study, the time of gene therapy a week later usual postnatal 14 days, but we show that therapeutic effect is also distinct in a naturally occurring mouse model of CNGA3 achromatopsia. The results provide us the illumination for future AAV5-based gene therapy for human CNGA3 achromatopsia.
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