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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Progress Towards Liver and Lung-Directed Gene Therapy with Helper- Dependent Adenoviral Vectors

Author(s): Nicola Brunetti-Pierri, Philip Ng.

Abstract:

Helper-dependent adenoviral vectors (HDAd) have several characteristics making them very attractive for human gene therapy. These vectors are completely devoid of viral coding sequences and are able to mediate high efficiency transduction in vivo to direct high level transgene expression with negligible chronic toxicity. Progress towards liver and lung directed gene therapy with HDAd as well as the current obstacles facing human applications and possible strategies to overcome these obstacles are discussed.

Keywords: Gene therapy, adenoviral vectors, helper-dependent adenoviral vectors, gutless, cystic fibrosis

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Article Details

VOLUME: 9
ISSUE: 5
Year: 2009
Page: [329 - 340]
Pages: 12
DOI: 10.2174/156652309789753310
Price: $58