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Titolo:
Synthesis of galactosyl compounds for targeted gene delivery
Autore:
Ren, T; Zhang, GS; Liu, DX;
Indirizzi:
Univ Pittsburgh, Sch Pharm, Dept Pharmaceut Sci, Pittsburgh, PA 15261 USA Univ Pittsburgh Pittsburgh PA USA 15261 eut Sci, Pittsburgh, PA 15261 USA
Titolo Testata:
BIOORGANIC & MEDICINAL CHEMISTRY
fascicolo: 11, volume: 9, anno: 2001,
pagine: 2969 - 2978
SICI:
0968-0896(200111)9:11<2969:SOGCFT>2.0.ZU;2-T
Fonte:
ISI
Lingua:
ENG
Soggetto:
HEPATIC ASIALOGLYCOPROTEIN RECEPTOR; POLY-L-LYSINE; CATIONIC LIPIDS; HIGH-AFFINITY; IN-VITRO; N-ACETYLGALACTOSAMINE; CLUSTER GALACTOSIDES; MEDIATED DELIVERY; HEPATOCYTES; LIVER;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
62
Recensione:
Indirizzi per estratti:
Indirizzo: Liu, DX Univ Pittsburgh, Sch Pharm, Dept Pharmaceut Sci, Pittsburgh, PA 15261 USA Univ Pittsburgh Pittsburgh PA USA 15261 Pittsburgh, PA 15261 USA
Citazione:
T. Ren et al., "Synthesis of galactosyl compounds for targeted gene delivery", BIO MED CH, 9(11), 2001, pp. 2969-2978

Abstract

Cell-specific DNA delivery offers a great potential for targeted gene therapy. Toward this end, we have synthesized a series of compounds carrying galactose residues as a targeting ligand for asialoglycoprotein receptors of hepatocytes and primary amine groups as a functional domain for DNA binding. Biological activity of these galactosyl compounds in DNA delivery was evaluated in HepG2 and BL-6 cells and compared with respect to the number of galactose residues as well as primary amine groups in each molecule. Transfection. experiments using a firefly luciferase gene as a reporter revealed that compounds with multivalent binding properties were more active in DNA delivery. An optimal transfection activity in HepG2 cells requires seven primary amine groups and a minimum of two galactose residues in each molecule. The transfection activity of compounds carrying multi-galactose residues can be inhibited by asialofetuin, a natural substrate for asialoglycoproteinreceptors of hepatocytes, suggesting that gene transfer by these galactosyl compounds is asialoglycoprotein receptor-mediated. These results provide direct evidence in support of our new strategy for the use of small and synthetic compounds for cell specific and targeted gene delivery. (C) 2001 Elsevier Science Ltd. All rights reserved.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 22/01/20 alle ore 18:48:06