Establishing an in vitro disease model for Fabry Disease using patient specific induced pluripotent stem cell-derived sensory neurons
Fabry disease (FD) is an X-linked lysosomal storage disorder caused by deficiency of the α-galactosidase A (GLA), leading to intracellular accumulations of globotriaosylceramide (Gb3). Acral burning pain, which can be triggered by heat, fever or physical activity is an early hallmark of FD and great...
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| Format: | Doctoral Thesis |
| Language: | English |
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2021
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| Online Access: | https://opus.bibliothek.uni-wuerzburg.de/frontdoor/index/index/docId/19970 http://nbn-resolving.de/urn:nbn:de:bvb:20-opus-199705 https://nbn-resolving.org/urn:nbn:de:bvb:20-opus-199705 https://doi.org/10.25972/OPUS-19970 https://opus.bibliothek.uni-wuerzburg.de/files/19970/Klein_Thomas_iPSC_Dissertation.pdf |
Internet
https://opus.bibliothek.uni-wuerzburg.de/frontdoor/index/index/docId/19970http://nbn-resolving.de/urn:nbn:de:bvb:20-opus-199705
https://nbn-resolving.org/urn:nbn:de:bvb:20-opus-199705
https://doi.org/10.25972/OPUS-19970
https://opus.bibliothek.uni-wuerzburg.de/files/19970/Klein_Thomas_iPSC_Dissertation.pdf