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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Detalles Bibliográficos
Autores principales: Han, Sang-oh, Li, Songtao, McCall, Angela, Arnson, Benjamin, Everitt, Jeffrey I., Zhang, Haoyue, Young, Sarah P., ElMallah, Mai K., Koeberl, Dwight D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6938806/
https://www.ncbi.nlm.nih.gov/pubmed/31909086
http://dx.doi.org/10.1016/j.omtm.2019.11.020

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