Low-Dose Gene Therapy for Murine PKU Using Episomal Naked DNA Vectors Expressing PAH from Its Endogenous Liver Promoter

• Autors principals: Hiu Man Grisch-Chan (Autor), Andrea Schlegel (Autor), Tanja Scherer (Autor), Gabriella Allegri (Autor), Raphael Heidelberger (Autor), Panagiota Tsikrika (Autor), Marco Schmeer (Autor), Martin Schleef (Autor), Cary O. Harding (Autor), Johannes Häberle (Autor), Beat Thöny (Autor)
• Format: Llibre
• Publicat: Elsevier, 2017-06-01T00:00:00Z.
• Matèries: article
• Accés en línia: Connect to this object online.

Limited duration of transgene expression, insertional mutagenesis, and size limitations for transgene cassettes pose challenges and risk factors for many gene therapy vectors. Here, we report on physiological expression of liver phenylalanine hydroxylase (PAH) by delivery of naked DNA/minicircle (MC...