Scheme 1.

a) Schematic illustration showing that two supramolecular nanoparticle (SMNP) vectors were developed for codelivery of Cas9/sgRNA‐plasmid and Donor‐RS1/GFP‐plasmid, enabling CRISPR/Cas9‐mediated knockin of RS1 gene in mouse retinas. After Cas9•sgRNA formation in vivo, CRISPR/Cas9‐mediated knockin of RS1 gene is carried out in two consecutive steps following the homology‐independent targeted integration (HITI) strategy. b) A self‐assembled synthetic strategy adopted for preparation of Cas9/sgRNA‐plasmid⊂SMNPs through stoichiometric mixing of Cas9/sgRNA‐plasmid and four SMNP molecular building blocks, i.e., CD‐PEI, Ad‐PAMAM, Ad‐PEG, and Ad‐PEG‐TAT. c) A self‐assembly strategy adopted for preparation of Donor‐RS1/GFP‐plasmid⊂SMNPs.