{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Chen G"],"funding":["NEI NIH HHS","NHLBI NIH HHS","NINDS NIH HHS","NIGMS NIH HHS"],"pagination":["974-980"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC6778035"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["14(10)"],"pubmed_abstract":["Delivery technologies for the CRISPR-Cas9 (CRISPR, clustered regularly interspaced short palindromic repeats) gene editing system often require viral vectors, which pose safety concerns for therapeutic genome editing<sup>1</sup>. Alternatively, cationic liposomal components or polymers can be used to encapsulate multiple CRISPR components into large particles (typically >100 nm diameter); however, such systems are limited by variability in the loading of the cargo. Here, we report the design of customizable synthetic nanoparticles for the delivery of Cas9 nuclease and a single-guide RNA (sgRNA) that enables the controlled stoichiometry of CRISPR components and limits the possible safety concerns in vivo. We describe the synthesis of a thin glutathione (GSH)-cleavable covalently crosslinked"],"journal":["Nature nanotechnology"],"pubmed_title":["A biodegradable nanocapsule delivers a Cas9 ribonucleoprotein complex for in vivo genome editing."],"pmcid":["PMC6778035"],"funding_grant_id":["R01 HL129785","R01 NS091540","R01 EY024995","R35 GM119644","UG3 NS111688","R01 HL143469"],"pubmed_authors":["Robertson S","Gong S","Suzuki M","Shahi PK","Chen G","Abdeen AA","Pattnaik BR","Saha K","Wang Y","Xie R"],"additional_accession":[]},"is_claimable":false,"name":"A biodegradable nanocapsule delivers a Cas9 ribonucleoprotein complex for in vivo genome editing.","description":"Delivery technologies for the CRISPR-Cas9 (CRISPR, clustered regularly interspaced short palindromic repeats) gene editing system often require viral vectors, which pose safety concerns for therapeutic genome editing<sup>1</sup>. Alternatively, cationic liposomal components or polymers can be used to encapsulate multiple CRISPR components into large particles (typically >100 nm diameter); however, such systems are limited by variability in the loading of the cargo. Here, we report the design of customizable synthetic nanoparticles for the delivery of Cas9 nuclease and a single-guide RNA (sgRNA) that enables the controlled stoichiometry of CRISPR components and limits the possible safety concerns in vivo. We describe the synthesis of a thin glutathione (GSH)-cleavable covalently crosslinked","dates":{"release":"2019-01-01T00:00:00Z","publication":"2019 Oct","modification":"2025-04-04T00:05:20.669Z","creation":"2020-05-22T12:16:01Z"},"accession":"S-EPMC6778035","cross_references":{"pubmed":["31501532"],"doi":["10.1038/s41565-019-0539-2"]}}