scholarly journals Long-Term Restoration of Thymidine Phosphorylase Function and Nucleoside Homeostasis Using Hematopoietic Gene Therapy in a Murine Model of Mitochondrial Neurogastrointestinal Encephalomyopathy

2016 ◽  
Vol 27 (9) ◽  
pp. 656-667 ◽  
Author(s):  
Javier Torres-Torronteras ◽  
Raquel Cabrera-Pérez ◽  
Ignasi Barba ◽  
Carme Costa ◽  
Noemí de Luna ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Murine Model ◽  
Thymidine Phosphorylase ◽  
Mitochondrial Neurogastrointestinal Encephalomyopathy

scholarly journals Thymidine phosphorylase is both a therapeutic and a suicide gene in a murine model of mitochondrial neurogastrointestinal encephalomyopathy

Gene Therapy ◽  
2014 ◽  
Vol 21 (7) ◽  
pp. 673-681 ◽  
Author(s):  
S López-Estévez ◽  
G Ferrer ◽  
J Torres-Torronteras ◽  
M J Mansilla ◽  
S Casacuberta-Serra ◽  
...  
Keyword(s):  
Murine Model ◽  
Thymidine Phosphorylase ◽  
Suicide Gene ◽  
Mitochondrial Neurogastrointestinal Encephalomyopathy

Long-term metabolic correction of Wilson’s disease in a murine model by gene therapy

2016 ◽  
Vol 64 (2) ◽  
pp. 419-426 ◽  
Author(s):  
Oihana Murillo ◽  
Daniel Moreno Luqui ◽  
Cristina Gazquez ◽  
Debora Martinez-Espartosa ◽  
Iñigo Navarro-Blasco ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Murine Model ◽  
Wilson’S Disease ◽  
Wilson's Disease ◽  
Metabolic Correction

scholarly journals Long-Term Sustained Effect of Liver-Targeted Adeno-Associated Virus Gene Therapy for Mitochondrial Neurogastrointestinal Encephalomyopathy

2018 ◽  
Vol 29 (6) ◽  
pp. 708-718 ◽  
Author(s):  
Javier Torres-Torronteras ◽  
Raquel Cabrera-Pérez ◽  
Ferran Vila-Julià ◽  
Carlo Viscomi ◽  
Yolanda Cámara ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Adeno Associated Virus ◽  
Mitochondrial Neurogastrointestinal Encephalomyopathy ◽  
Virus Gene ◽  
Sustained Effect

Long-term efficacy and safety of hematopoietic stem cell gene therapy mediated by lentiviral vectors in the murine model of Wiskott-Aldrich syndrome

2008 ◽  
Vol 40 (2) ◽  
pp. 256-257
Author(s):  
Marita Bosticardo ◽  
Francesco Marangoni ◽  
Michela Locci ◽  
Elena Draghici ◽  
Samantha Scaramuzza ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Murine Model ◽  
Efficacy And Safety ◽  
Hematopoietic Stem ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

scholarly journals Waning efficacy in a long-term AAV-mediated gene therapy study in the murine model of Krabbe disease

2021 ◽  
Author(s):  
Gregory J. Heller ◽  
Michael S. Marshall ◽  
Yazan Issa ◽  
Jeffrey N. Marshall ◽  
Duc Nguyen ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Murine Model ◽  
Krabbe Disease
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