CRISPR-LbCpf1 prevents choroidal neovascularization in a mouse model of age-related macular degeneration

The CRISPR endonuclease LbCpf1 is reported to have greater efficiency and specificity than Cas9. Here, the authors use LbCpf1 to target the angiogenesis-related genes VEGF and HIF1a, and show that delivery of the nuclease using AAV9 is effective in mouse models of macular degeneration.

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Autores principales: Taeyoung Koo, Sung Wook Park, Dong Hyun Jo, Daesik Kim, Jin Hyoung Kim, Hee-Yeon Cho, Jeungeun Kim, Jeong Hun Kim, Jin-Soo Kim
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2018
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Acceso en línea:https://doaj.org/article/25757b2b16cb4f498da30b72b2a47f63
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Sumario:The CRISPR endonuclease LbCpf1 is reported to have greater efficiency and specificity than Cas9. Here, the authors use LbCpf1 to target the angiogenesis-related genes VEGF and HIF1a, and show that delivery of the nuclease using AAV9 is effective in mouse models of macular degeneration.