Minimally invasive and selective hydrodynamic gene therapy of liver segments in the pig and human

被引:60
作者
Khorsandi, S. E. [1 ]
Bachellier, P. [1 ,2 ]
Weber, J. C. [1 ,2 ]
Greget, M. [2 ]
Jaeck, D. [2 ]
Zacharoulis, D. [3 ]
Rountas, C. [3 ]
Helmy, S. [1 ,4 ]
Helmy, A. [4 ]
Al-Waracky, M. [4 ]
Salama, H. [4 ]
Jiao, L. [1 ]
Nicholls, J. [1 ]
Davies, A. J. [1 ]
Levicar, N. [1 ]
Jensen, S. [1 ]
Habib, N. [1 ]
机构
[1] Univ London Imperial Coll Sci Technol & Med, Fac Med, Hammersmith Hosp, London W12 0NN, England
[2] Hosp Hautpierre, Fdn Transplantat, Strasbourg, France
[3] Larissa Univ, Sch Med, Larisa, Greece
[4] Cairo Univ, Fac Med, Cairo, Egypt
关键词
hydrodynamic gene therapy; human; pig;
D O I
10.1038/sj.cgt.7701119
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
This paper highlights our experience of the transfer of hydrodynamic gene therapy (HGT) from the large animal, the pig, into clinical practice. The modification of balloon catheters and the development of a minimally invasive technique to allow selective isolation of liver segments for HGT in the large animal and human are described. Finally, our preliminary results from a phase I clinical study of HGT for thrombopoietin (TPO) in cirrhotic patients with thrombocytopenia are discussed. Based on these provisional data, minimally invasive selective HGT of liver segments appears to be technically safe, but further work is required to optimize the efficiency of gene transfer in order to achieve clinical benefit.
引用
收藏
页码:225 / 230
页数:6
相关论文
共 11 条
[1]   Pig liver gene therapy by noninvasive interventionist catheterism [J].
Alino, S. F. ;
Herrero, M. J. ;
Noguera, I. ;
Dasi, F. ;
Sanchez, M. .
GENE THERAPY, 2007, 14 (04) :334-343
[2]   Hydrodynamics-based transfection of the liver: entrance into hepatocytes of DNA that causes expression takes place very early after injection [J].
Andrianaivo, F ;
Lecocq, M ;
De Coninck, SW ;
Wattiaux, R ;
Jadot, M .
JOURNAL OF GENE MEDICINE, 2004, 6 (08) :877-883
[3]  
Budker V, 1996, GENE THER, V3, P593
[4]   Hydrodynamic liver gene transfer mechanism involves transient sinusoidal blood stasis and massive hepatocyte endocytic vesicles [J].
Crespo, A ;
Peydró, A ;
Dasí, F ;
Benet, M ;
Calvete, JJ ;
Revert, F ;
Aliño, SF .
GENE THERAPY, 2005, 12 (11) :927-935
[5]   Development of catheter-based procedures for transducing the isolated rabbit liver with plasmid DNA [J].
Eastman, SJ ;
Baskin, KM ;
Hodges, BL ;
Chu, QM ;
Gates, A ;
Dreusicke, R ;
Anderson, S ;
Scheule, RK .
HUMAN GENE THERAPY, 2002, 13 (17) :2065-2077
[6]  
Habib N., 2004, GENE THERAPY MOL BIO, V8, P1
[7]   Hyrodynamics-based procedure involves transient hyperpermeability in the hepatic cellular membrane: implication of a nonspecific process in efficient intracellular gene delivery [J].
Kobayashi, N ;
Nishikawa, M ;
Hirata, K ;
Takakura, Y .
JOURNAL OF GENE MEDICINE, 2004, 6 (05) :584-592
[8]   Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA [J].
Liu, F ;
Song, YK ;
Liu, D .
GENE THERAPY, 1999, 6 (07) :1258-1266
[9]   Long-term and therapeutic-level hepatic gene expression of human factor IX after naked plasmid transfer in vivo [J].
Miao, CH ;
Thompson, AR ;
Loeb, K ;
Ye, X .
MOLECULAR THERAPY, 2001, 3 (06) :947-957
[10]   Naked plasmid DNA transfer to the porcine liver using rapid injection with large volume [J].
Yoshino, H. ;
Hashizume, K. ;
Kobayashi, E. .
GENE THERAPY, 2006, 13 (24) :1696-1702