Endosomal Escape and Delivery of CRISPR/Cas9 Genome Editing Machinery Enabled by Nanoscale Zeolitic Imidazolate Framework
AuthorsAlsaiari, Shahad K.
Alyami, Mram Z.
Aleisa, Fajr A
Khashab, Niveen M.
KAUST DepartmentAdvanced Membranes and Porous Materials Research Center
Biological and Environmental Sciences and Engineering (BESE) Division
Chemical Science Program
Physical Science and Engineering (PSE) Division
Smart Hybrid Materials (SHMs) lab
Online Publication Date2017-12-27
Print Publication Date2018-01-10
Permanent link to this recordhttp://hdl.handle.net/10754/626437
MetadataShow full item record
AbstractCRISPR/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 employing CRISPR/Cas9 machinery. The nanoscale CC-ZIFs are biocompatible and easily scaled-up offering excellent loading capacity and controlled co-delivery of intact Cas9 protein and sgRNA.
CitationAlsaiari SK, Patil S, Alyami M, Alamoudi K, Aleisa fajr, et al. (2017) Endosomal Escape and Delivery of CRISPR/Cas9 Genome Editing Machinery Enabled by Nanoscale Zeolitic Imidazolate Framework. Journal of the American Chemical Society. Available: http://dx.doi.org/10.1021/jacs.7b11754.
PublisherAmerican Chemical Society (ACS)
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