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.

Object Details

• Location: Deutsche Nationalbibliothek Frankfurt am Main
• Extent: Online-Ressource
• Language: Englisch

References and Relationships

• Bibliographic citation: 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

Contributors, Places and Time

• Creator: Büeler, H.

Further information

• DOI: 10.1515/BC.1999.078
• URN: urn:nbn:de:101:1-2406111612201.381779489482
• Rights: Open Access; Der Zugriff auf das Objekt ist unbeschränkt möglich.
• Last update: 14.08.2025, 10:49 AM CEST