Low-Dose Gene Therapy for Murine PKU Using Episomal Naked DNA Vectors Expressing PAH from Its Endogenous Liver Promoter-Reference-Cited by-同舟云学术

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

Published:2017-06 Issue: Volume:7 Page:339-349
ISSN:2162-2531
Container-title:Molecular Therapy - Nucleic Acids
language:en
Short-container-title:Molecular Therapy - Nucleic Acids

Author:

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

Funder

Children’s Research Centre Zurich

Rare Disease Initiative of the University of Zurich (radiz)

Wolfermann Nägeli Stiftung

Swiss National Science Foundation

Publisher

Elsevier BV

Subject

Drug Discovery,Molecular Medicine

Reference52 articles.

1. Phenylketonuria;Blau;Lancet,2010

2. Long-term correction of murine phenylketonuria by viral gene transfer: liver versus muscle;Thöny;J. Inherit. Metab. Dis.,2010

3. Pahhph-5: a mouse mutant deficient in phenylalanine hydroxylase;McDonald;Proc. Natl. Acad. Sci. USA,1990

4. Gene therapy returns to centre stage;Naldini;Nature,2015

5. Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas;Nault;Nat. Genet.,2015

Cited by 20 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Therapeutic liver cell transplantation to treat a genetic liver defect;2024-06-27

2. Current Advances and Material Innovations in the Search for Novel Treatments of Phenylketonuria;Advanced Healthcare Materials;2024-05-31

3. Continuous directed evolution of a compact CjCas9 variant with broad PAM compatibility;Nature Chemical Biology;2023-09-21

4. State‐of‐the‐art 2023 on gene therapy for phenylketonuria;Journal of Inherited Metabolic Disease;2023-08-03

5. Intrabiliary infusion of naked DNA vectors targets periportal hepatocytes in mice;Molecular Therapy - Methods & Clinical Development;2022-12