CasRx-mediated RNA targeting prevents choroidal neovascularization in a mouse model of age-related macular degeneration

Zhou, C.; Hu, X.; Tang, C.; Liu, W.; Wang, S.; Zhou, Y.; Zhao, Q.; Bo, Q.; Shi, L.; Sun, X.; Zhou, H.; Yang, H.

doi:10.1101/2020.02.18.955286

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bioRxiv · 10.1101/2020.02.18.955286

CasRx-mediated RNA targeting prevents choroidal neovascularization in a mouse model of age-related macular degeneration

Zhou, C.·Hu, X.·Tang, C.·Liu, W.·Wang, S.·Zhou, Y.·Zhao, Q.·Bo, Q.·Shi, L.·Sun, X.·Zhou, H.·Yang, H.

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Abstract

The smallest Cas13 family protein, CasRx, has a high cleavage activity and targeting specificity, offering attractive opportunity for therapeutic applications. Here we report that delivery of CasRx by adeno-associated virus via intravitreal injection could efficiently knockdown Vegfa transcripts and significantly reduce the area of laser-induced choroidal neovascularization in a mouse model of age-related macular degeneration. Thus, RNA-targeting CRISPR system could be used for in vivo gene therapy.

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Zhou, C., Hu, X., Tang, C., Liu, W., Wang, S., Zhou, Y., Zhao, Q., Bo, Q., Shi, L., Sun, X., Zhou, H., Yang, H.. 2020-02-20. CasRx-mediated RNA targeting prevents choroidal neovascularization in a mouse model of age-related macular degeneration. https://doi.org/10.1101/2020.02.18.955286

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