¹Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA

Correspondence: Dr P Ng, Department of Molecular and Human Genetics, Baylor College of Medicine, One Baylor Plaza, T619, Houston, TX 77030, USA. E-mail: png@bcm.tmc.edu

Received 8 August 2007; Revised 16 January 2008; Accepted 16 January 2008; Published online 21 February 2008.

Abstract

Preclinical studies in small and large animal models using helper-dependent adenoviral vectors (HDAds) have generated promising results for the treatment of genetic diseases. However, clinical translation is complicated by the dose-dependent, capsid-mediated acute toxic response following systemic vector injection. With the advancements in vectorology, a better understanding of vector-mediated toxicity, and improved delivery methods, HDAds may emerge as an important vector for gene therapy of genetic diseases and this report highlights recent progress and prospects in this field.