Current Gene Therapy

Ignacio Anegon
Director INSERM UMR 1064-Center for Research in Transplantation and Immunology
CHU de Nantes. 30, boulevard
Nantes
France

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Rate Limiting Steps of AAV Transduction and Implications for Human Gene Therapy

Author(s): S. Sanlioglu, M. M. Monick, G. Luleci, G. W. Hunninghake, J. F. Engelhardt.

Abstract:

Despite the fact that adeno-associated virus type 2 (AAV2) is an extremely attractive gene therapy vector, its application has been limited to certain tissues such as muscle and the brain. In an attempt to broaden the array of target organs for this vector, molecular studies on the mechanism(s) of AAV transduction have expanded over the past several years. These studies have led to the development of innovative strategies capable of overcoming intracellular barriers to AAV2 transduction. The basis of these technologic breakthroughs has stemmed from a better understanding of the molecular processes that control AAV entry and intracellular trafficking to the nucleus. This review will focus on the identification of molecular components important for recombinant AAV (rAAV) transduction while highlighting the techniques used to discover them and potential clinical application of research findings.

Keywords: AAV Transduction, Human Gene Therapy, Recombinant AAV (rAA V), Viral Binding, Heparan sulfate proteoglycan, Nuclear Transport, Viral Uncoating, Nuclear Gene Conversion

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Article Details

VOLUME: 1
ISSUE: 2
Year: 2001
Page: [137 - 147]
Pages: 11
DOI: 10.2174/1566523013348788
Price: $58