scholarly journals Gene therapy using a novel G6PC-S298C variant enhances the long-term efficacy for treating glycogen storage disease type Ia

2020 ◽  
Vol 527 (3) ◽  
pp. 824-830
Author(s):  
Lisa Zhang ◽  
Cheol Lee ◽  
Irina Arnaoutova ◽  
Javier Anduaga ◽  
Matthew F. Starost ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage Disease Type ◽  
Glycogen Storage ◽  
Disease Type ◽  
Term Efficacy ◽  
Type Ia

scholarly journals Long-Term Efficacy Following Readministration of an Adeno-Associated Virus Vector in Dogs with Glycogen Storage Disease Type Ia

2012 ◽  
Vol 23 (4) ◽  
pp. 407-418 ◽  
Author(s):  
Amanda Demaster ◽  
Xiaoyan Luo ◽  
Sarah Curtis ◽  
Kyha D. Williams ◽  
Dustin J. Landau ◽  
...  
Keyword(s):  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage Disease Type ◽  
Glycogen Storage ◽  
Disease Type ◽  
Virus Vector ◽  
Adeno Associated Virus ◽  
Term Efficacy ◽  
Type Ia

Long-term safety and efficacy of AAV gene therapy in the canine model of glycogen storage disease type Ia

2018 ◽  
Vol 41 (6) ◽  
pp. 977-984 ◽  
Author(s):  
Young Mok Lee ◽  
Thomas J. Conlon ◽  
Andrew Specht ◽  
Kirsten E. Coleman ◽  
Laurie M. Brown ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage Disease Type ◽  
Canine Model ◽  
Glycogen Storage ◽  
Disease Type ◽  
Safety And Efficacy ◽  
Type Ia

scholarly journals Downregulation of pathways implicated in liver inflammation and tumorigenesis of glycogen storage disease type Ia mice receiving gene therapy

2017 ◽  
Vol 26 (10) ◽  
pp. 1890-1899 ◽  
Author(s):  
Goo-Young Kim ◽  
Joon Hyun Kwon ◽  
Jun-Ho Cho ◽  
Lisa Zhang ◽  
Brian C. Mansfield ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage Disease Type ◽  
Glycogen Storage ◽  
Disease Type ◽  
Liver Inflammation ◽  
Type Ia
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