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Titolo:
Adeno associated viral vectors for gene transfer and gene therapy
Autore:
Bueler, H;
Indirizzi:
Univ Zurich, Inst Mol Biol, CH-8057 Zurich, Switzerland Univ Zurich Zurich Switzerland CH-8057 Biol, CH-8057 Zurich, Switzerland
Titolo Testata:
BIOLOGICAL CHEMISTRY
fascicolo: 6, volume: 380, anno: 1999,
pagine: 613 - 622
SICI:
1431-6730(199906)380:6<613:AAVVFG>2.0.ZU;2-R
Fonte:
ISI
Lingua:
ENG
Soggetto:
RECOMBINANT ADENOASSOCIATED VIRUS; SITE-SPECIFIC INTEGRATION; DNA-BINDING PROTEIN; EXPRESSION IN-VIVO; AAV VECTOR; REP PROTEINS; FACTOR-IX; INTRAMUSCULAR INJECTION; PERSISTENT EXPRESSION; TRANSGENE EXPRESSION;
Keywords:
gene therapy; gene transfer; recombinant adeno-associated virus; retargeting; transcriptional regulation;
Tipo documento:
Review
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
106
Recensione:
Indirizzi per estratti:
Indirizzo: Bueler, H Univ Zurich, Inst Mol Biol, Winterthurerstr 190, CH-8057 Zurich,Switzerland Univ Zurich Winterthurerstr 190 Zurich Switzerland CH-8057 land
Citazione:
H. Bueler, "Adeno associated viral vectors for gene transfer and gene therapy", BIOL CHEM, 380(6), 1999, pp. 613-622

Abstract

Adeno-associated virus (AAV) is a defective, nonpathogenic human parvovirus that depends for growth on coinfection with a helper adenovirus or herpesvirus. Recombinant adeno-associated viruses (rAAVs) have attracted considerable interest as vectors for gene therapy. In contrast to other gene delivery systems, rAAVs lack all viral genes and show long-term gene expression in vivo without immune response or toxicity. Over the past few years, many applications of rAAVs as therapeutic agents have demonstrated the utility of this vector system for long-lasting genetic modification and gene therapyin preclinical models of human disease. New production methods have increased rAAV vector titers and eliminated contamination by adenovirus. In addition, vectors for regulatable gene expression and vectors retargeted to different cells have been engineered. These advancements are expected to accelerate and facilitate further animal model studies, providing validation for use of rAAVs in human clinical trials.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 01/06/20 alle ore 22:46:58