Fig. 4: In vivo evaluation of AAV6-SpCas9 transduction of ocular epithelial tropism in marmoset model. | Gene Therapy

Fig. 4: In vivo evaluation of AAV6-SpCas9 transduction of ocular epithelial tropism in marmoset model.

From: Multiplex viral tropism assay in complex cell populations with single-cell resolution

Fig. 4: In vivo evaluation of AAV6-SpCas9 transduction of ocular epithelial tropism in marmoset model.

A AAV virus titer in the marmoset cornea epithelial tissue was quantified by qPCR using specific probe and primers and normalized to diploid copies. Comparison between control left cornea and treated right cornea was analyzed by Student t test (two-tailed) using the software GraphPad Prism 9. *p < 0.05. B Cas9 mRNA expression level was quantified in extracted RNA from the marmoset cornea epithelial tissue and normalized using GAPDH. Comparison between control left cornea and treated right cornea was analyzed by Student t test (two-tailed) using the software GraphPad Prism 9. *p < 0.05. C Immunohistochemistry staining of marmoset1 cornea epithelial tissue. Presence of AAV viral antigen (first row) and SpCas9 cargo protein (third row) was detected in the right cornea epithelial layer (co-stained by CK3 antibody) but absent in the untreated left cornea epithelial layer (second and fourth row). D Immunohistochemistry staining of marmoset2 cornea epithelial tissue. Presence of AAV viral antigen (first row) and SpCas9 cargo protein (third row) was detected in the right cornea epithelial layer (co-stained by CK3 antibody) but absent in the untreated left cornea epithelial layer (second and fourth row).

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