Adeno-Associated Viral Vectors for Gene Transfer and Gene Therapy

Abstract: Adeno-associated virus (AAV) is a defective, non-pathogenic human parvovirus that depends for growth on coinfection with a helper adenovirus or herpes virus. 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 therapy in 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.

Standort
Deutsche Nationalbibliothek Frankfurt am Main
Umfang
Online-Ressource
Sprache
Englisch

Erschienen in
Adeno-Associated Viral Vectors for Gene Transfer and Gene Therapy ; volume:380 ; number:6 ; year:1999 ; pages:613-622
Biological chemistry ; 380, Heft 6 (1999), 613-622

Urheber
Büeler, H.

DOI
10.1515/BC.1999.078
URN
urn:nbn:de:101:1-2406111612201.381779489482
Rechteinformation
Open Access; Der Zugriff auf das Objekt ist unbeschränkt möglich.
Letzte Aktualisierung
14.08.2025, 10:49 MESZ

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Beteiligte

  • Büeler, H.

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