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

Pompe disease is caused by the deficiency of lysosomal acid α-glucosidase (GAA). It is expected that gene therapy to replace GAA with adeno-associated virus (AAV) vectors will be less effective early in life because of the rapid loss of vector genomes. AAV2/8-LSPhGAA (3 × 10(10) vector genomes [vg]/...

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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Han, Sang-oh, Li, Songtao, McCall, Angela, Arnson, Benjamin, Everitt, Jeffrey I., Zhang, Haoyue, Young, Sarah P., ElMallah, Mai K., Koeberl, Dwight D.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2019
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6938806/
https://ncbi.nlm.nih.gov/pubmed/31909086
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.11.020
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