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AAV Gene Therapy Utilizing Glycosylation-Independent Lysosomal Targeting Tagged GAA in the Hypoglossal Motor System of Pompe Mice

Pompe disease is caused by mutations in the gene encoding the lysosomal glycogen-metabolizing enzyme, acid-alpha glucosidase (GAA). Tongue myofibers and hypoglossal motoneurons appear to be particularly susceptible in Pompe disease. Here we used intramuscular delivery of adeno-associated virus serot...

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Detalles Bibliográficos
Autores principales:	Doyle, Brendan M., Turner, Sara M.F., Sunshine, Michael D., Doerfler, Phillip A., Poirier, Amy E., Vaught, Lauren A., Jorgensen, Marda L., Falk, Darin J., Byrne, Barry J., Fuller, David 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/PMC6807287/ https://www.ncbi.nlm.nih.gov/pubmed/31660421 http://dx.doi.org/10.1016/j.omtm.2019.08.009

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