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Comparisons of Infant and Adult Mice Reveal Age Effects for Liver Depot Gene Therapy in Pompe Disease
Molecular Therapy — Methods & Clinical Development
◽
10.1016/j.omtm.2019.11.020
◽
2020
◽
Vol 17
◽
pp. 133-142
◽
Cited By ~ 2
Author(s):
Sang-oh Han
◽
Songtao Li
◽
Angela McCall
◽
Benjamin Arnson
◽
Jeffrey I. Everitt
◽
...
Keyword(s):
Gene Therapy
◽
Pompe Disease
◽
Age Effects
◽
Adult Mice
Download Full-text
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Cited By
References
Comparisons of infant and adult mice reveal age effects for liver depot gene therapy in Pompe disease
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2019.11.165
◽
2020
◽
Vol 129
(2)
◽
pp. S69-S70
Author(s):
Sang-oh Han
◽
Angela McCall
◽
Benjamin Arnson
◽
Songtao Li
◽
Jeffrey I. Everitt
◽
...
Keyword(s):
Gene Therapy
◽
Pompe Disease
◽
Age Effects
◽
Adult Mice
Download Full-text
Muscle‐directed gene therapy corrects Pompe disease and uncovers species‐specific GAA immunogenicity
EMBO Molecular Medicine
◽
10.15252/emmm.202113968
◽
2021
◽
Author(s):
Michelle Eggers
◽
Charles H Vannoy
◽
Jianyong Huang
◽
Pravinkumar Purushothaman
◽
Jacqueline Brassard
◽
...
Keyword(s):
Gene Therapy
◽
Pompe Disease
◽
Species Specific
Download Full-text
275. Ex Vivo Lentiviral Gene Therapy as a Cure for Murine Pompe Disease
Molecular Therapy
◽
10.1016/s1525-0016(16)34610-x
◽
2013
◽
Vol 21
◽
pp. S105
Keyword(s):
Gene Therapy
◽
Pompe Disease
◽
Ex Vivo
◽
Lentiviral Gene Therapy
Download Full-text
Correction of Multiple Striated Muscles in Murine Pompe Disease Through Adeno-associated Virus–mediated Gene Therapy
Molecular Therapy
◽
10.1038/mt.2008.133
◽
2008
◽
Vol 16
(8)
◽
pp. 1366-1371
◽
Cited By ~ 50
Author(s):
Baodong Sun
◽
Sarah P Young
◽
Ping Li
◽
Chunhui Di
◽
Talmage Brown
◽
...
Keyword(s):
Gene Therapy
◽
Pompe Disease
◽
Striated Muscles
◽
Adeno Associated Virus
Download Full-text
A phase 1 study of gene therapy with ACTUS-101 in late-onset Pompe disease
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2019.11.227
◽
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
Download Full-text
239. Immunomodulation Is Necessary to Achieve Stable Expression after Retroviral Vector-Mediated Liver-Directed Gene Therapy to Adult Mice with MPS I
Molecular Therapy
◽
10.1016/j.ymthe.2005.06.242
◽
2005
◽
Vol 11
◽
pp. S94-S95
Keyword(s):
Gene Therapy
◽
Retroviral Vector
◽
Stable Expression
◽
Adult Mice
◽
Mps I
Download Full-text
Lentiviral Stem Cell Gene Therapy for Pompe Disease
Journal of Neuromuscular Diseases
◽
10.3233/jnd-159055
◽
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
Download Full-text
Clinical Gene Therapy Trials for Pompe Disease
Muscle Gene Therapy
◽
10.1007/978-3-030-03095-7_44
◽
2019
◽
pp. 759-774
Author(s):
Cristina Liberati
◽
Stephanie Salabarria
◽
Manuela Corti
◽
Barry J. Byrne
Keyword(s):
Gene Therapy
◽
Pompe Disease
◽
Clinical Gene Therapy
Download Full-text
Preclinical development of , an investigational liver-directed AAV gene therapy for the treatment of Pompe disease
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2019.11.029
◽
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
Download Full-text
Assessment of toxicity and biodistribution of recombinant AAV8 vector–mediated immunomodulatory gene therapy in mice with Pompe disease
Molecular Therapy — Methods & Clinical Development
◽
10.1016/j.omtm.2019.05.001
◽
2019
◽
Vol 13
◽
pp. 493
Author(s):
Gensheng Wang
◽
Sarah P. Young
◽
Deeksha Bali
◽
Julie Hutt
◽
Songtao Li
◽
...
Keyword(s):
Gene Therapy
◽
Pompe Disease
◽
Immunomodulatory Gene
Download Full-text
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