Hepatocyte transplantation as a treatment for glycogen storage disease type 1a

M. Muraca, G. Gerunda, D. Neri, M. T. Vilei, A. Granato, P. Feltracco, M. Meroni, G. Giron, A. B. Burlina

Research output: Contribution to journalArticlepeer-review

Abstract

Treatment of many inherited disorders of hepatic metabolism is still challenging. Hepatocyte transplantation was done in a 47-year-old woman who had glycogen storage disease type 1a and severe fasting hypoglycaemia. 2 billion viable hepatocytes were infused via an indwelling portal-vein catheter, followed by a triple immunosuppression regimen with mycophenolate mofetil, tacrolimus, and steroids. 9 months after transplantation, on only tacrolimus, she eats a normal diet and can fast for 7 h without experiencing hypoglycaemia. Our results show that hepatocyte transplantation might be an alternative to liver transplantation in glycogen storage disease type 1a.

Original languageEnglish
Pages (from-to)317-318
Number of pages2
JournalLancet
Volume359
Issue number9303
DOIs
Publication statusPublished - Jan 26 2002

ASJC Scopus subject areas

  • Medicine(all)

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