Progress and prospects: gene therapy for genetic diseases with helper-dependent adenoviral vectors.
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.
Brunetti-Pierri
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Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.
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D000256:Adenoviridae / Q000235:genetics*; D005544:Forecasting; D030342:Genetic Diseases, Inborn / Q000628:therapy*; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D005822:Genetic Vectors / Q000008:administration & dosage*; D006378:Helper Viruses / Q000235:genetics*; D006801:Humans; D008099:Liver / Q000378:metabolism; D008168:Lung / Q000378:metabolism; D014161:Transduction, Genetic / Q000379:methods*