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Article

In vitro study of encapsulation therapy for Fabry disease using genetically engineered CHO cell line.

Cell transplantation - 1 Jan 2002

Naganawa Y, Ohsugi K, Kase R, Date I, Sakuraba H, Sakuragawa N

Abstract excerpt

Fabry disease is an X-linked recessive disorder caused by a deficiency of the lysosomal hydrolase alpha-galactosidase A (alpha-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 human alpha-gal produced in CHO cells or in human fibroblasts is currently being evaluated in clinical trials...

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Source

Identifiers

PMID: 12162373

Genes

CTH

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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