In Vitro Gene Transfer with a Novel Galactosylated Spermine Bolaamphiphile
详细信息 查看全文
- 作者：Jé ; rô ; me Gaucheron ; Catherine Santaella ; and Pierre Vierling
- 刊名：Bioconjugate Chemistry
- 出版年：2001
- 出版时间：July 2001
- 年：2001
- 卷：12
- 期：4
- 页码：569 - 575
- 全文大小：139K
- 年卷期：v.12,no.4(July 2001)
- ISSN：1520-4812
文摘
We describe the synthesis of a 
-galacto-
-spermine bolaamphiphile (GalSper) and report on the genetransfer mediated with lipoplexes it forms either when used alone or in conjunction with DOPE orwith DOGS (Transfectam). Lipofection with GalSper was investigated with human HepG2 or murineBNL-CL2 hepatocytes expressing the asialo-glycoprotein (ASGP) receptor, which displays a high affinityfor galactosyl residues, or with A549 cells which do not express ASGP. Although lower luciferaseexpression levels in BNL-CL2 and in HepG2 cells were obtained with GalSper/DOPE N/P 2.5 lipoplexesas compared with control DOGS/DOPE N/P 2.5 particles or with the more positively charged N/P 5particles (yet through a different mechanism), specific receptor-mediated endocytosis of DNA can beachieved with this targeted cationic GalSper bolaamphiphile presenting a single galactose residue.The present work suggests that GalSper-based DNA formulations appear as promising syntheticvectors for specific gene delivery to ASGP(+) cells.
-galacto--spermine bolaamphiphile (GalSper) and report on the genetransfer mediated with lipoplexes it forms either when used alone or in conjunction with DOPE orwith DOGS (Transfectam). Lipofection with GalSper was investigated with human HepG2 or murineBNL-CL2 hepatocytes expressing the asialo-glycoprotein (ASGP) receptor, which displays a high affinityfor galactosyl residues, or with A549 cells which do not express ASGP. Although lower luciferaseexpression levels in BNL-CL2 and in HepG2 cells were obtained with GalSper/DOPE N/P 2.5 lipoplexesas compared with control DOGS/DOPE N/P 2.5 particles or with the more positively charged N/P 5particles (yet through a different mechanism), specific receptor-mediated endocytosis of DNA can beachieved with this targeted cationic GalSper bolaamphiphile presenting a single galactose residue.The present work suggests that GalSper-based DNA formulations appear as promising syntheticvectors for specific gene delivery to ASGP(+) cells.