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

scholarly journals Lentiviral Stem Cell Gene Therapy for Pompe Disease

2015 ◽  
Vol 2 (s1) ◽  
pp. S64-S64
Author(s):  
Qiushi Liang ◽  
Merel Stok ◽  
Yvette van Helsdingen ◽  
Guus van der Velden ◽  
Ed Jacobs ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Pompe Disease ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

Clinical Gene Therapy Trials for Pompe Disease

2019 ◽  
pp. 759-774
Author(s):  
Cristina Liberati ◽  
Stephanie Salabarria ◽  
Manuela Corti ◽  
Barry J. Byrne
Keyword(s):  
Gene Therapy ◽  
Pompe Disease ◽  
Clinical Gene Therapy

Preclinical development of , an investigational liver-directed AAV gene therapy for the treatment of Pompe disease

2020 ◽  
Vol 129 (2) ◽  
pp. S22-S23
Author(s):  
Sean M. Armour ◽  
Jayme M.L. Nordin ◽  
Helena Costa Verdera ◽  
Pauline Sellier ◽  
George M. Preston ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Pompe Disease ◽  
Preclinical Development

Generation of Plasmid DNA Expressing Species-Specific Horse VEGF164 and FGF2 Factors for Gene Therapy

2016 ◽  
Vol 6 (4) ◽  
pp. 550-553 ◽  
Author(s):  
Y. A. Litvin ◽  
E. Yu Zakirova ◽  
M. N. Zhuravleva ◽  
A. A. Rizvanov
Keyword(s):  
Gene Therapy ◽  
Plasmid Dna ◽  
Species Specific

scholarly journals 382. Correction of Autophagic Dysregulation in Pompe Disease Following Gene Therapy

2016 ◽  
Vol 24 ◽  
pp. S152
Author(s):  
Angela L. McCall ◽  
Sylvia G. Stankov ◽  
Darin J. Falk ◽  
Barry J. Byrne
Keyword(s):  
Gene Therapy ◽  
Pompe Disease
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