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

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

Volume: 9 Issue: 5

Author(s): Nicola Brunetti-Pierri and Philip Ng

Affiliation:

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

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.

Cite this article as:

Brunetti-Pierri Nicola and Ng Philip, Progress Towards Liver and Lung-Directed Gene Therapy with Helper- Dependent Adenoviral Vectors, Current Gene Therapy 2009; 9 (5) . https://dx.doi.org/10.2174/156652309789753310