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. 2023 Jul 18;9:73. doi: 10.1038/s41421-023-00554-y

Fig. 1. In vivo gene editing and disease treatment using hypercompact Un1Cas12f1 in a mouse model of tyrosinaemia.

Fig. 1

a Schematic map of three Cas12f1 orthologs (AsCas12f1, SpCas12f1 and Un1Cas12f1). b In vitro editing efficiency of three Cas12f1 orthologs for target sites of mouse Trp53 gene in N2a cells. c In vitro editing efficiency for mouse Hpd and Tyr genes with Un1Cas12f1 in N2a cells. d Workflow of embryonic injection of Un1Cas12f1 mRNA and gRNA to generate gene-edited mice. e Gene editing efficiency in mice after embryonic injection of Un1Cas12f1 mRNA and gRNA. f Schematic map of AAV-CjCas9 and AAV-Un1Cas12f1. g Procedure of gene therapy with AAV-CjCas9 and AAV-Un1Cas12f1 in a mouse model of tyrosinaemia. h Deep-sequencing results of gene editing outcomes for liver DNA after Cas12f1 treatment. i Survival curve for tyrosinaemia mice treated with or without AAV-CjCas9 and AAV-Un1Cas12f1. j Mouse weight analysis after treatment with or without AAV-CjCas9 and AAV-Un1Cas12f1. k HPD staining results of liver from tyrosinaemia mice treated with or without AAV-CjCas9 and AAV-Un1Cas12f1. All mice were injected with AAV at the age of 8 weeks. Data are represented as means ± SEM. Scale bars, 200 μm.