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7T MRI Predicts Amelioration of Neurodegeneration in the Brain after AAV Gene Therapy
GM1 gangliosidosis (GM1) is a fatal neurodegenerative lysosomal storage disease that occurs most commonly in young children, with no effective treatment available. Long-term follow-up of GM1 cats treated by bilateral thalamic and deep cerebellar nuclei (DCN) injection of adeno-associated virus (AAV)...
Autores principales: | Gray-Edwards, Heather L., Maguire, Anne S., Salibi, Nouha, Ellis, Lauren E., Voss, Taylor L., Diffie, Elise B., Koehler, Jey, Randle, Ashley N., Taylor, Amanda R., Brunson, Brandon L., Denney, Thomas S., Beyers, Ronald J., Gentry, Atoska S., Gross, Amanda L., Batista, Ana R., Sena-Esteves, Miguel, Martin, Douglas R. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6962699/ https://www.ncbi.nlm.nih.gov/pubmed/31970203 http://dx.doi.org/10.1016/j.omtm.2019.11.023 |
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