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. 2021 Jan 27;108(2):295–308. doi: 10.1016/j.ajhg.2021.01.006

Figure 4.

Figure 4

Allele-specific editing in mouse photoreceptors

(A) (Top) a scheme of the experimental timeline is depicted; (middle) AAV2/8 vectors expressing the WT or VQRHF1 SpCas9 under the control of IRBP promoter are schematized; (bottom) AAV2/8 vectors expressing the gRNA (gRNA1, gRNA5, or scramble) and the GFP under the control of RHO promoter.

(B) Indels frequency determined by NGS in retinae injected with effector vectors.

(C) Representation of indels scored in retinae treated with gRNA1 or gRNA5 AAV vector coupled to appropriate AAV-SpCas9 vector. Nucleotides inserted or deleted are reported on the right.

(D) Downregulation of RHO transcript coding for p.Pro347Ser RHO in retinae injected with effector vectors with respect to retinae treated with scramble vectors. The averages are depicted with a bar. ∗p < 0.05.

(E) Rhodopsin localization was investigated at P50 in retinae injected with effector vectors using 4D2 and 1D4 antibodies against rhodopsin. Representative images are shown. Scale bar represents 10 μm. Zoomed areas of photoreceptors are shown on the side. OS, outer segment; IS, inner segment; ONL, outer nuclear layer.