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| Liver cell transplantation for Crigler-Najjar syndrome type Ⅰ： Update and perspectives | |
| 引用本文： | Lysy PA,Najimi M,Stephenne X,Bourgois A,Smets F,Sokal EM. Liver cell transplantation for Crigler-Najjar syndrome type Ⅰ： Update and perspectives[J]. World journal of gastroenterology : WJG, 2008, 14(22): 3464-3470. DOI: 10.3748/wjg.14.3464 |
| 作者姓名： | Lysy PA Najimi M Stephenne X Bourgois A Smets F Sokal EM |
| 作者单位： | Philippe A Lysy（Universite Catholique de Louvain, Cliniques Universitaires Saint Luc, HPED Department, PEDI Unit, Laboratory of Pediatric Hepatology and Cell Therapy, Brussels B-1200, Belgium） ;Mustapha Najimi（Universite Catholique de Louvain, Cliniques Universitaires Saint Luc, HPED Department, PEDI Unit, Laboratory of Pediatric Hepatology and Cell Therapy, Brussels B-1200, Belgium） ;Xavier Stephenne（Universite Catholique de Louvain, Cliniques Universitaires Saint Luc, HPED Department, PEDI Unit, Laboratory of Pediatric Hepatology and Cell Therapy, Brussels B-1200, Belgium） ;Annick Bourgois（Universite Catholique de Louvain, Cliniques Universitaires Saint Luc, HPED Department, PEDI Unit, Laboratory of Pediatric Hepatology and Cell Therapy, Brussels B-1200, Belgium） ;Francoise Smets（Universite Catholique de Louvain, Cliniques Universitaires Saint Luc, HPED Department, PEDI Unit, Laboratory of Pediatric Hepatology and Cell Therapy, Brussels B-1200, Belgium） ;Etienne M Sokal（Universite Catholique de Louvain, Cliniques Universitaires Saint Luc, HPED Department, PEDI Unit, Laboratory of Pediatric Hepatology and Cell Therapy, Brussels B-1200, Belgium） ; |
| 摘 要： | Liver cell transplantation is an attractive technique to treat liver-based inborn errors of metabolism. The feasibility and efficacy of the procedure has been demonstrated, leading to medium term partial metabolic control of various diseases. Crigler-Najjar is the paradigm of such diseases in that the host liver is lacking one function with an otherwise normal parenchyma. The patient is at permanent risk for irreversible brain damage. The goal of liver cell transplantation is to reduce serum bilirubin levels within safe limits and to alleviate phototherapy requirements to improve quality of life. Preliminary data on Gunn rats, the rodent model of the disease, were encouraging and have led to successful clinical trials. Herein we report on two additional patients and describe the current limits of the technique in terms of durability of the response as compared to alternative therapeutic procedures. We discuss the future developments of the technique and new emerging perspectives. |
| 关 键 词： | 肝移植 细胞治疗 先天性代谢故障 肝再生 |
Liver cell transplantation for Crigler-Najjar syndrome type I: update and perspectives |
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| Lysy Philippe-A,Najimi Mustapha,Stephenne Xavier,Bourgois Annick,Smets Francoise,Sokal Etienne-M. Liver cell transplantation for Crigler-Najjar syndrome type I: update and perspectives[J]. World journal of gastroenterology : WJG, 2008, 14(22): 3464-3470. DOI: 10.3748/wjg.14.3464 | |
| Authors: | Lysy Philippe-A Najimi Mustapha Stephenne Xavier Bourgois Annick Smets Francoise Sokal Etienne-M |
| Affiliation: | Universite Catholique de Louvain, Cliniques Universitaires Saint Luc, HPED Department, PEDI Unit, Laboratory of Pediatric Hepatology and Cell Therapy, Brussels B-1200, Belgium |
| Abstract: | Liver cell transplantation is an attractive technique to treat liver-based inborn errors of metabolism. The feasibility and efficacy of the procedure has been demonstrated, leading to medium term partial metabolic control of various diseases. CriglerNajjar is the paradigm of such diseases in that the host liver is lacking one function with an otherwise normal parenchyma. The patient is at permanent risk for irreversible brain damage. The goal of liver cell transplantation is to reduce serum bilirubin levels within safe limits and to alleviate phototherapy requirements to improve quality of life. Preliminary data on Gunn rats, the rodent model of the disease, were encouraging and have led to successful clinical trials. Herein we report on two additional patients and describe the current limits of the technique in terms of durability of the response as compared to alternative therapeutic procedures. We discuss the future developments of the technique and new emerging perspectives. |
| Keywords: | Hepatocyte transplantation Cell therapy Inborn error of metabolism Crigler-Najjar Liver regeneration Animal models |
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