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

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...

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Detalles Bibliográficos
Autores principales: Grisch-Chan, Hiu Man, Schlegel, Andrea, Scherer, Tanja, Allegri, Gabriella, Heidelberger, Raphael, Tsikrika, Panagiota, Schmeer, Marco, Schleef, Martin, Harding, Cary O., Häberle, Johannes, Thöny, Beat
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2017
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5423318/
https://www.ncbi.nlm.nih.gov/pubmed/28624210
http://dx.doi.org/10.1016/j.omtn.2017.04.013

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