Inclusion of the Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element Enhances AAV2-Driven Transduction of Mouse and Human Retina

The woodchuck hepatitis virus posttranscriptional regulatory element (WPRE) has been included in the transgene cassette of adeno-associated virus (AAV) in several gene therapy clinical trials, including those for inherited retinal diseases. However, the extent to which WPRE increases transgene expre...

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Bibliographic Details
Main Authors: Maria I. Patrício, Alun R. Barnard, Harry O. Orlans, Michelle E. McClements, Robert E. MacLaren
Format: Article
Language:English
Published: Elsevier 2017-03-01
Series:Molecular Therapy: Nucleic Acids
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253116303742