Gene Therapy for Metabolic Diseases of the Liver

被引：0
作者：
Kittichai Promrat
George Y. Wu
Catherine H. Wu
机构：
[1] University of Connecticut Health Center,Department of Medicine, Division of Gastroenterology
[2] University of Connecticut Health Center,Hepatology
来源：
BioDrugs | 2000年 / 13卷
关键词：
Gene Therapy; Adenoviral Vector; Hammerhead Ribozyme; Nonviral Vector; Asialoglycoprotein Receptor;
D O I：
暂无
中图分类号：
学科分类号：
摘要：
Significant advances have been made in the field of liver-directed gene therapy. Many diseases are potential targets for gene therapy, including diseases that have exclusive liver involvement and those with systemic manifestations as a result of defective protein synthesis from the liver. Examples are Crigler-Najjar syndrome type 1, α1-antitrypsin deficiency and haemophilia A and B.
引用
收藏
页码：177 / 188
页数：11
相关论文
共 177 条
  • [1] Kalpana G.V.(1999)Retroviral vectors for liver-directed gene therapy Semin Liver Dis 19 27-37
  • [2] Kafri T.(1997)Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors Nat Genet 17 314-7
  • [3] Blomer U.(1991)Long term improvement of hypercholesterolemia after Science 254 1802-5
  • [4] Peterson D.A.(1994) gene therapy in LDLR-deficient rabbits Nat Get 6 335-41
  • [5] Chowdhury J.R.(1998)Successful Liver Transplant Surg 4 78-88
  • [6] Grossman M.(1998) gene therapy directed to liver in a patient with familial hypercholesterolemia Gene Ther 5 552-5
  • [7] Gupta S.(1998)Long term reduction of serum bilirubin levels in Gunn rats by retroviral gene transfer in vivo Hepatology 28 707-16
  • [8] Grossman M.(1999)Retroviral gene transfer to the liver in vivo during tri-iodothyronine induced hyperplasia Hum Gene Ther 10 911-22
  • [9] Raper S.E.(1998)Hepatocyte growth factor induces hepatocyte proliferation Hum Gene Ther 9 1449-56
  • [10] Kosarsky K.(1998) and allows for efficient retroviral-mediated gene transfer in mice J Biol Chem 273 11954-61
12345678910