Publication
CRISPR/Cas9‐mediated genome editing: from basic research to translational medicine
| dc.contributor.author | Jacinto, Filipe | |
| dc.contributor.author | Link, Wolfgang | |
| dc.contributor.author | Ferreira, B I | |
| dc.date.accessioned | 2020-03-17T16:40:07Z | |
| dc.date.available | 2020-03-17T16:40:07Z | |
| dc.date.issued | 2020 | |
| dc.description.abstract | The recent development of the CRISPR/Cas9 system as an efficient and accessible programmable genome-editing tool has revolutionized basic science research. CRISPR/Cas9 system-based technologies have armed researchers with new powerful tools to unveil the impact of genetics on disease development by enabling the creation of precise cellular and animal models of human diseases. The therapeutic potential of these technologies is tremendous, particularly in gene therapy, in which a patient-specific mutation is genetically corrected in order to treat human diseases that are untreatable with conventional therapies. However, the translation of CRISPR/Cas9 into the clinics will be challenging, since we still need to improve the efficiency, specificity and delivery of this technology. In this review, we focus on several in vitro, in vivo and ex vivo applications of the CRISPR/Cas9 system in human disease-focused research, explore the potential of this technology in translational medicine and discuss some of the major challenges for its future use in patients. | pt_PT |
| dc.description.sponsorship | Spanish Ministry of Science, Innovation and Universities RTI2018-094629-B-I00 ; 748585 LPCC-NRS/Terry Fox grants; RTI2018-094629-B-I00 | pt_PT |
| dc.description.version | info:eu-repo/semantics/publishedVersion | pt_PT |
| dc.identifier.doi | https://doi.org/10.1111/jcmm.14916 | pt_PT |
| dc.identifier.issn | 1582-1838 | |
| dc.identifier.uri | http://hdl.handle.net/10400.1/13599 | |
| dc.language.iso | eng | pt_PT |
| dc.peerreviewed | yes | pt_PT |
| dc.publisher | Wiley | pt_PT |
| dc.relation | Characterizing the clinical relevance of TRIB2 mediated resistance to PI3K pathway inhibition in colon and breast cancer | |
| dc.relation.publisherversion | https://onlinelibrary.wiley.com/doi/full/10.1111/jcmm.14916 | pt_PT |
| dc.rights.uri | http://creativecommons.org/licenses/by/4.0/ | pt_PT |
| dc.subject | CRISPR | pt_PT |
| dc.subject | Gene therapy | pt_PT |
| dc.subject | Genome editing | pt_PT |
| dc.subject | Translational medicine | pt_PT |
| dc.title | CRISPR/Cas9‐mediated genome editing: from basic research to translational medicine | pt_PT |
| dc.type | journal article | |
| dspace.entity.type | Publication | |
| oaire.awardTitle | Characterizing the clinical relevance of TRIB2 mediated resistance to PI3K pathway inhibition in colon and breast cancer | |
| oaire.awardURI | info:eu-repo/grantAgreement/FCT/5876/UID%2FBIM%2F04773%2F2013/PT | |
| oaire.awardURI | info:eu-repo/grantAgreement/FCT/OE/SFRH%2FBPD%2F100434%2F2014/PT | |
| oaire.citation.title | Journal of Cellular and Molecular Medicine | pt_PT |
| oaire.fundingStream | 5876 | |
| oaire.fundingStream | OE | |
| person.familyName | Jacinto | |
| person.familyName | Link | |
| person.familyName | ferreira | |
| person.givenName | Filipe | |
| person.givenName | Wolfgang | |
| person.givenName | Bibiana | |
| person.identifier | 803637 | |
| person.identifier.ciencia-id | 991A-568B-78ED | |
| person.identifier.ciencia-id | 6910-952E-242A | |
| person.identifier.ciencia-id | A311-E925-09C5 | |
| person.identifier.orcid | 0000-0002-3340-5165 | |
| person.identifier.orcid | 0000-0003-4772-9395 | |
| person.identifier.scopus-author-id | 35368713800 | |
| project.funder.identifier | http://doi.org/10.13039/501100001871 | |
| project.funder.identifier | http://doi.org/10.13039/501100001871 | |
| project.funder.name | Fundação para a Ciência e a Tecnologia | |
| project.funder.name | Fundação para a Ciência e a Tecnologia | |
| rcaap.rights | openAccess | pt_PT |
| rcaap.type | article | pt_PT |
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