title: Hepatocyte transplantation as a treatment for glycogen storage disease type 1a.
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.
authors.forename: Maurizio
authors.initials: M
authors.lastname: Muraca
authors.forename: Giorgio
authors.initials: G
authors.lastname: Gerunda
authors.forename: Daniele
authors.initials: D
authors.lastname: Neri
authors.forename: Maria-Teresa
authors.initials: MT
authors.lastname: Vilei
authors.forename: Anna
authors.initials: A
authors.lastname: Granato
authors.forename: Paolo
authors.initials: P
authors.lastname: Feltracco
authors.forename: Muzio
authors.initials: M
authors.lastname: Meroni
authors.forename: Gianpiero
authors.initials: G
authors.lastname: Giron
authors.forename: Alberto B
authors.initials: AB
authors.lastname: Burlina
mesh_terms: D002408:Catheters, Indwelling; D017690:Cell Transplantation; D005260:Female; D005500:Follow-Up Studies; D005953:Glycogen Storage Disease Type I / Q000175:diagnosis / Q000601:surgery*; D006801:Humans; D016031:Liver Transplantation; D008875:Middle Aged; D011169:Portal Vein; D016896:Treatment Outcome