In Vitro Study of Encapsulation Therapy for Fabry Disease Using Genetically Engineered CHO Cell Line
Fabry disease is an X-linked recessive disorder caused by a deficiency of the lysosomal hydrolase α-galac-tosidase A (α-gal). The deficiency of this enzyme leads to the systemic deposition of ceramide trihexoside (CTH) in various tissues and organs. Enzyme replacement using IV doses of recombinant h...
| Main Authors: | , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
SAGE Publishing
2002-05-01
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| Series: | Cell Transplantation |
| Online Access: | https://doi.org/10.3727/000000002783985846 |