Skip to main content
Fig. 5 | Journal of Nanobiotechnology

Fig. 5

From: Nanoparticles-mediated CRISPR-Cas9 gene therapy in inherited retinal diseases: applications, challenges, and emerging opportunities

Fig. 5

In vivo delivery of the CRISPR-Cas9 machinery using SMNPs. A, B Schemes of the self-assembly of SMNPs loaded with plasmid DNA encoding Cas9/gRNA or donor-RS1/GFP. C A schematic presentation showing the intravitreal injection of SMNPs loaded with the indicated plasmids, resulting in the delivery of CRISPR-Cas9 machinery to express RS1 and GFP proteins in retinal layers. D Fundus photography (left) and optical coherence tomography (right) images showing GFP signals and retinal structure after SMNP-mediated gene delivery. E H&E and immunohistochemistry staining of GFP-positive cells in the retinal layers. F Electrophoretogram showing PCR amplification of the right-arm junction (617 bp) and left-arm junction (748 bp). G Sanger sequencing of the genome-donor boundaries showing the effective CRISPR-Cas9-mediated knock-in of RS1/GFP genes in vivo. Data reproduced from our previous study [119]

Back to article page