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EnglishIn vivo application of the CRISPR-Cas9 technology is still limited by unwanted Cas9 genomic cleavages. Long-term expression of Cas9 increases the number of genomic loci non-specifically cleaved by the nuclease. Here we develop a Self-Limiting Cas9 circuit for Enhanced Safety and specificity (SLiCES) which consists of an expression unit for Streptococcus pyogenes Cas9 (SpCas9), a self-targeting sgRNA and a second sgRNA targeting a chosen genomic locus. The self-limiting circuit results in increased genome editing specificity by controlling Cas9 levels. For its in vivo utilization, we next integrate SLiCES into a lentiviral delivery system (lentiSLiCES) via circuit inhibition to achieve viral particle production. Upon delivery into target cells, the lentiSLiCES circuit switches on to edit the intended genomic locus while simultaneously stepping up its own neutralization through SpCas9 inactivation. By preserving target cells from residual nuclease activity, our hit and go system increases safety margins for genome editing.
Hit and go CAS9 delivered through a lentiviral based self-limiting circuit / Petris, Gianluca; Casini, Antonio; Montagna, Claudia; Lorenzin, Francesca; Prandi, Davide; Romanel, Alessandro; Zasso, Jacopo; Conti, Luciano; Demichelis, Francesca; Cereseto, Anna. - In: NATURE COMMUNICATIONS. - ISSN 2041-1723. - ELETTRONICO. - 8(2017), p. 15334.
| Titolo: | Hit and go CAS9 delivered through a lentiviral based self-limiting circuit |
| Autori: | Petris, Gianluca; Casini, Antonio; Montagna, Claudia; Lorenzin, Francesca; Prandi, Davide; Romanel, Alessandro; Zasso, Jacopo; Conti, Luciano; Demichelis, Francesca; Cereseto, Anna |
| Autori Unitn: | |
| Titolo del periodico: | NATURE COMMUNICATIONS |
| Anno di pubblicazione: | 2017 |
| Codice identificativo Scopus: | 2-s2.0-85019953441 |
| Codice identificativo Pubmed: | 28530235 |
| Codice identificativo ISI: | WOS:000401907100001 |
| Digital Object Identifier (DOI): | http://dx.doi.org/10.1038/ncomms15334 |
| Handle: | http://hdl.handle.net/11572/188564 |
| Citazione: | Hit and go CAS9 delivered through a lentiviral based self-limiting circuit / Petris, Gianluca; Casini, Antonio; Montagna, Claudia; Lorenzin, Francesca; Prandi, Davide; Romanel, Alessandro; Zasso, Jacopo; Conti, Luciano; Demichelis, Francesca; Cereseto, Anna. - In: NATURE COMMUNICATIONS. - ISSN 2041-1723. - ELETTRONICO. - 8(2017), p. 15334. |
| Appare nelle tipologie: | 03.1 Articolo su rivista (Journal article) |
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| ncomms15334.pdf | Articolo principale | Versione editoriale (Publisher’s layout) |  | Open Access Visualizza/Apri |
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