P.F508del editing in cells from cystic fibrosis patients

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dc.contributor.authorLaura Tammaro
dc.contributor.authorChiara Barriga
dc.contributor.authorSamuele Turrini
dc.contributor.authorMaria Favia
dc.contributor.authorMonica Gallucci
dc.contributor.authorElena Beccia
dc.contributor.authorSilvia Recchia
dc.date.accessioned2025-08-20T04:04:16Z
dc.date.available2025-08-20T04:04:16Z
dc.date.issued2021-01-01
dc.description.abstractThis study demonstrates the correction of the p.F508del mutation in the CFTR gene using CRISPR/Cas9 in CFTE29o‑ cells and induced pluripotent stem cells derived from cystic fibrosis patients. Editing efficiency was quantified by measuring both NHEJ and HDR frequencies. In CFTE29o‑ cells, NHEJ ranged from 1.25 % to 2.54 % of alleles and HDR reached up to 1.42 %. In patient‑derived iPSCs, NHEJ varied from 5.5 % to 12.13 %, while the best HDR efficiency was 2.38 %. The results reveal that gene editing of the p.F508del mutation via CRISPR/Cas9 is a relatively rare event, suggesting that subsequent cell selection and cultivation are necessary for practical application., en
dc.identifier.citationTammaro, L.; Barriga, C.; Turrini, S.; et al. (2021). Front. Genome Ed., 3:638492. https://doi.org/10.3389/fgeed.2021.638492en
dc.identifier.doi10.3389/fgeed.2021.638492
dc.identifier.urihttps://doi.org/10.3389/fgeed.2021.638492
dc.identifier.urihttps://nur.nu.edu.kz/handle/123456789/9647
dc.language.isoen
dc.publisherFrontiers
dc.relation.ispartofFrontiers in Genome Editingen
dc.rightsOpen accessen
dc.sourceFrontiers in Genome Editing, 3, 638492, (2021)en
dc.subjectcystic fibrosisen
dc.subjectgenome editingen
dc.subjectCRISPRen
dc.subjectCFTRen
dc.titleP.F508del editing in cells from cystic fibrosis patientsen
dc.typeJournal Articleen

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