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

• Main Authors: Maria I. Patrício (Author), Alun R. Barnard (Author), Harry O. Orlans (Author), Michelle E. McClements (Author), Robert E. MacLaren (Author)
• Format: Knjiga
• Izdano: Elsevier, 2017-03-01T00:00:00Z.
• Teme: article
• Online dostop: Connect to this object online.

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...