Genetic repair of a human induced pluripotent cell line from patient with Dutch-type cerebral amyloid angiopathy
Dutch-type cerebral amyloid angiopathy (D-CAA), also known as hereditary cerebral haemorrhage with amyloidosis-Dutch type (HCHWA-D), is an autosomal dominant disorder caused by a G to C transversion in codon 693 of the amyloid precursor protein (APP) that results in a Gln-to-Glu amino acid substitution. CRISPR-Cas9 editing was used for genetic correction of the mutation in a human induced pluripotent stem cell (hiPSC-) line established previously. The isogenic hiPSCs generated showed typical pluripotent stem cell morphology, expressed all markers of undifferentiated state, displayed a normal k... Mehr ...
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| Dokumenttyp: | Artikel |
| Erscheinungsdatum: | 2023 |
| Reihe/Periodikum: | Stem Cell Research, Vol 71, Iss , Pp 103180- (2023) |
| Verlag/Hrsg.: |
Elsevier
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| Schlagwörter: | Biology (General) / QH301-705.5 |
| Sprache: | Englisch |
| Permalink: | https://search.fid-benelux.de/Record/base-26630058 |
| Datenquelle: | BASE; Originalkatalog |
| Powered By: | BASE |
| Link(s) : | https://doi.org/10.1016/j.scr.2023.103180 |
Dutch-type cerebral amyloid angiopathy (D-CAA), also known as hereditary cerebral haemorrhage with amyloidosis-Dutch type (HCHWA-D), is an autosomal dominant disorder caused by a G to C transversion in codon 693 of the amyloid precursor protein (APP) that results in a Gln-to-Glu amino acid substitution. CRISPR-Cas9 editing was used for genetic correction of the mutation in a human induced pluripotent stem cell (hiPSC-) line established previously. The isogenic hiPSCs generated showed typical pluripotent stem cell morphology, expressed all markers of undifferentiated state, displayed a normal karyotype and had the capacity to differentiate into the three germ layers.