| dc.contributor.author | NOURSHARGH, S | |
| dc.contributor.author | Santiago-Fernández, O | |
| dc.contributor.author | Osorio, F G | |
| dc.contributor.author | Quesada, V | |
| dc.contributor.author | Rodríguez, F | |
| dc.contributor.author | Basso, S | |
| dc.contributor.author | Maeso, D | |
| dc.contributor.author | Rolas, L | |
| dc.contributor.author | Barkaway, A | |
| dc.contributor.author | Folgueras, A R | |
| dc.contributor.author | Freije, J M P | |
| dc.contributor.author | López-Otín, C | |
| dc.date.accessioned | 2019-03-15T15:18:11Z | |
| dc.date.available | 2018-12-18 | |
| dc.date.available | 2019-03-15T15:18:11Z | |
| dc.date.issued | 2019-02-18 | |
| dc.identifier.issn | 1078-8956 | |
| dc.identifier.uri | https://qmro.qmul.ac.uk/xmlui/handle/123456789/56282 | |
| dc.description.abstract | CRISPR/Cas9-based therapies hold considerable promise for the treatment of genetic diseases. Among these, Hutchinson–Gilford progeria syndrome, caused by a point mutation in the LMNA gene, stands out as a potential candidate. Here, we explore the efficacy of a CRISPR/Cas9-based approach that reverts several alterations in Hutchinson–Gilford progeria syndrome cells and mice by introducing frameshift mutations in the LMNA gene. | en_US |
| dc.description.sponsorship | Wellcome Trust (No. 098291/Z/12/Z to S.N.) | en_US |
| dc.language.iso | en | en_US |
| dc.publisher | Nature Research | en_US |
| dc.relation.ispartof | Nature Medicine | |
| dc.title | Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria syndrome | en_US |
| dc.type | Article | en_US |
| dc.identifier.doi | https://doi.org/10.1038/s41591-018-0338-6 | |
| pubs.notes | Not known | en_US |
| pubs.publication-status | Accepted | en_US |
| dcterms.dateAccepted | 2018-12-18 | |
| rioxxterms.funder | Default funder | en_US |
| rioxxterms.identifier.project | Default project | en_US |
| qmul.funder | Mode and dynamics of neutrophil transmigration in vivo: Mechanisms and implications to pathological inflammation::Wellcome Trust | en_US |
