scholarly journals Corrigendum to “Assessment of toxicity and biodistribution of recombinant AAV8 vector–mediated immunomodulatory gene therapy in mice with Pompe disease”

2015 ◽  
Vol 2 ◽  
pp. 15002 ◽  
Author(s):  
Gensheng Wang ◽  
Sarah P Young ◽  
Deeksha Bali ◽  
Julie Hutt ◽  
Songtao Li ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Pompe Disease ◽  
Immunomodulatory Gene

scholarly journals Assessment of toxicity and biodistribution of recombinant AAV8 vector–mediated immunomodulatory gene therapy in mice with Pompe disease

2014 ◽  
Vol 1 ◽  
pp. 14018 ◽  
Author(s):  
Gensheng Wang ◽  
Sarah P Young ◽  
Deeksha Bali ◽  
Julie Hutt ◽  
Songtao Li ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Pompe Disease ◽  
Immunomodulatory Gene

scholarly journals Immunomodulatory Gene Therapy Prevents Antibody Formation and Lethal Hypersensitivity Reactions in Murine Pompe Disease

2010 ◽  
Vol 18 (2) ◽  
pp. 353-360 ◽  
Author(s):  
Baodong Sun ◽  
Michael D Kulis ◽  
Sarah P Young ◽  
Amy C Hobeika ◽  
Songtao Li ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Pompe Disease ◽  
Antibody Formation ◽  
Hypersensitivity Reactions ◽  
Immunomodulatory Gene

scholarly journals Muscle‐directed gene therapy corrects Pompe disease and uncovers species‐specific GAA immunogenicity

2021 ◽  
Author(s):  
Michelle Eggers ◽  
Charles H Vannoy ◽  
Jianyong Huang ◽  
Pravinkumar Purushothaman ◽  
Jacqueline Brassard ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Pompe Disease ◽  
Species Specific

scholarly journals Correction of Multiple Striated Muscles in Murine Pompe Disease Through Adeno-associated Virus–mediated Gene Therapy

2008 ◽  
Vol 16 (8) ◽  
pp. 1366-1371 ◽  
Author(s):  
Baodong Sun ◽  
Sarah P Young ◽  
Ping Li ◽  
Chunhui Di ◽  
Talmage Brown ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Pompe Disease ◽  
Striated Muscles ◽  
Adeno Associated Virus

scholarly journals Comparisons of Infant and Adult Mice Reveal Age Effects for Liver Depot Gene Therapy in Pompe Disease

2020 ◽  
Vol 17 ◽  
pp. 133-142 ◽  
Author(s):  
Sang-oh Han ◽  
Songtao Li ◽  
Angela McCall ◽  
Benjamin Arnson ◽  
Jeffrey I. Everitt ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Pompe Disease ◽  
Age Effects ◽  
Adult Mice

A phase 1 study of gene therapy with ACTUS-101 in late-onset Pompe disease

2020 ◽  
Vol 129 (2) ◽  
pp. S91
Author(s):  
Dwight Koeberl ◽  
Edward C. Smith ◽  
Laura E. Case ◽  
Sam Hopkins ◽  
Martin K. Childers ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Pompe Disease ◽  
Late Onset ◽  
Phase 1 ◽  
Phase 1 Study
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