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...

ver descrição completa

Na minha lista:
Detalhes bibliográficos
Publicado no:Mol Ther Nucleic Acids
Main Authors: 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: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2017
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5423318/
https://ncbi.nlm.nih.gov/pubmed/28624210
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2017.04.013
Tags: Adicionar Tag
Sem tags, seja o primeiro a adicionar uma tag!

Registos relacionados