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Progress and prospects: gene therapy for genetic diseases with helper-dependent adenoviral vectors

Gene Therapy volume 15, pages 553–560 (2008)Cite this article

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.

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

    N Brunetti-Pierri & P Ng

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  1. N Brunetti-Pierri
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  2. P Ng
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Correspondence to P Ng.

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Brunetti-Pierri, N., Ng, P. Progress and prospects: gene therapy for genetic diseases with helper-dependent adenoviral vectors. Gene Ther 15, 553–560 (2008). https://doi.org/10.1038/gt.2008.14

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