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J Am Chem Soc ; 140(1): 143-146, 2018 01 10.
Artículo en Inglés | MEDLINE | ID: mdl-29272114

RESUMEN

CRISPR/Cas9 is a combined protein (Cas9) and an engineered single guide RNA (sgRNA) genome editing platform that offers revolutionary solutions to genetic diseases. It has, however, a double delivery problem owning to the large protein size and the highly charged RNA component. In this work, we report the first example of CRISPR/Cas9 encapsulated by nanoscale zeolitic imidazole frameworks (ZIFs) with a loading efficiency of 17% and enhanced endosomal escape promoted by the protonated imidazole moieties. The gene editing potential of CRISPR/Cas9 encapsulated by ZIF-8 (CC-ZIFs) is further verified by knocking down the gene expression of green fluorescent protein by 37% over 4 days. The nanoscale CC-ZIFs are biocompatible and easily scaled-up offering excellent loading capacity and controlled codelivery of intact Cas9 protein and sgRNA.


Asunto(s)
Sistemas CRISPR-Cas/fisiología , Endosomas/metabolismo , Edición Génica , Imidazoles/química , Nanopartículas/química , Zeolitas/química , Animales , Células CHO , Cricetulus , Tamaño de la Partícula
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