scholarly journals 1094. A Novel Replication Competent, but Packaging Deficient Adenoviral(Ad) Vector [E1+, 100K-]hGAA for High Level hGAA Expression and Reduced Toxicity in Gene Therapy of Glycogen Storage Disease II (GSD-II)

2005 ◽  
Vol 11 ◽  
pp. S421-S422
Keyword(s):  
Gene Therapy ◽  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage ◽  
Reduced Toxicity ◽  
High Level ◽  
Gsd Ii

scholarly journals Liver-directed gene therapy for murine glycogen storage disease type Ib

2017 ◽  
Vol 26 (22) ◽  
pp. 4395-4405 ◽  
Author(s):  
Joon Hyun Kwon ◽  
Young Mok Lee ◽  
Jun-Ho Cho ◽  
Goo-Young Kim ◽  
Javier Anduaga ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage Disease Type ◽  
Glycogen Storage ◽  
Disease Type

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

scholarly journals Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage Disease

2019 ◽  
Vol 15 ◽  
pp. 383-391 ◽  
Author(s):  
Hye-Ri Kang ◽  
Monika Gjorgjieva ◽  
Stephanie N. Smith ◽  
Elizabeth D. Brooks ◽  
Zelin Chen ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage ◽  
Hepatic Tumors ◽  
Canine Models
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