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Titolo:
Nonviral gene delivery to the lateral ventricles in rat brain: Initial evidence for widespread distribution and expression in the central nervous system
Autore:
Hecker, JG; Hall, LL; Irion, VR;
Indirizzi:
Univ Calif Davis, Sch Med, Dept Anesthesiol, Davis, CA 95616 USA Univ Calif Davis Davis CA USA 95616 Dept Anesthesiol, Davis, CA 95616 USA
Titolo Testata:
MOLECULAR THERAPY
fascicolo: 3, volume: 3, anno: 2001,
pagine: 375 - 384
SICI:
1525-0016(200103)3:3<375:NGDTTL>2.0.ZU;2-H
Fonte:
ISI
Lingua:
ENG
Soggetto:
IN-VIVO; ADENOASSOCIATED VIRUS; HEAT-SHOCK; RECOMBINANT ADENOVIRUS; CEREBROSPINAL-FLUID; NONHUMAN-PRIMATES; IMMUNE-RESPONSE; MOUSE-BRAIN; TRANSFECTION; THERAPY;
Keywords:
DNA; gene transfer; GFP; luciferase; Hsp70; transfection; formulation; cationic lipid; in vivo; neurons; CNS;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
55
Recensione:
Indirizzi per estratti:
Indirizzo: Hecker, JG Univ Calif Davis, Sch Med, Dept Anesthesiol, Neursci Bldg,1515 Newton Court,Room 411, Davis, CA 95616 USA Univ Calif Davis Neursci Bldg,1515 Newton Court,Room 411 Davis CA USA 95616
Citazione:
J.G. Hecker et al., "Nonviral gene delivery to the lateral ventricles in rat brain: Initial evidence for widespread distribution and expression in the central nervous system", MOL THER, 3(3), 2001, pp. 375-384

Abstract

The use of DNA for nonviral gene expression depends on several factors. These include (i) delivery and accessibility to the targeted tissue, (ii) protection from extracellular degradation, (iii) sufficient uptake by cells ofinterest, and (iv) protection from intracellular degradation to allow translation of adequate levels of intracellular or secreted proteins. As an initial step in demonstrating the feasibility of nonviral, cationic lipid-mediated gene therapy, we present evidence for the successful delivery and expression of heat shock protein Hsp70 and reporter gene enzymes in the centralnervous system (CNS) of the rat after injection into the lateral ventricle. Gene delivery is accomplished using optimized formulations of plasmid DNA, which have been complexed with the cationic lipid MLRI. Results from DNA vectors encoding for green fluorescent protein (GFP), luciferase, and Hsp70are reported. Standard immunofluorescent methods were used to demonstrate widespread expression of the reporter proteins and of Hsp70. Stereology analysis has been completed on three coronal sections, which illustrates the distribution of expression along the longitudinal axis. These initial findings support the further development of nonviral, lipid-mediated gene delivery technology for transient expression of protective, intracellular proteinsand represent an important step leading to in vivo studies to identify potential clinical benefits.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 18/01/20 alle ore 21:37:43