Progress and prospects: gene therapy for genetic diseases with helper-dependent adenoviral vectors
- 21 February 2008
- journal article
- review article
- Published by Springer Science and Business Media LLC in Gene Therapy
- Vol. 15 (8), 553-560
- https://doi.org/10.1038/gt.2008.14
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.Keywords
This publication has 50 references indexed in Scilit:
- One-year Expression From High-capacity Adenoviral Vectors in the Brains of Animals With Pre-existing Anti-adenoviral Immunity: Clinical ImplicationsMolecular Therapy, 2007
- The Influence of Blood on In Vivo Adenovirus Bio-distribution and TransductionMolecular Therapy, 2007
- Adenovirus-Platelet Interaction in Blood Causes Virus Sequestration to the Reticuloendothelial System of the LiverJournal of Virology, 2007
- Innate Immune Response to Adenoviral Vectors Is Mediated by both Toll-Like Receptor-Dependent and -Independent PathwaysJournal of Virology, 2007
- Adenovirus Infection Triggers a Rapid, MyD88-Regulated Transcriptome Response Critical to Acute-Phase and Adaptive Immune Responses In VivoJournal of Virology, 2007
- Preferential Activation of Toll-Like Receptor Nine by CD46-Utilizing AdenovirusesJournal of Virology, 2007
- Intrahepatic injection of adenovirus reduces inflammation and increases gene transfer and therapeutic effect in miceHepatology, 2006
- Improved Hepatic Transduction, Reduced Systemic Vector Dissemination, and Long-Term Transgene Expression by Delivering Helper-Dependent Adenoviral Vectors into the Surgically Isolated Liver of Nonhuman PrimatesHuman Gene Therapy, 2006
- Readministration of helper-dependent adenovirus to mouse lungGene Therapy, 2006
- Regulatable Gutless Adenovirus Vectors Sustain Inducible Transgene Expression in the Brain in the Presence of an Immune Response against AdenovirusesJournal of Virology, 2006