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)...

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Detalles Bibliográficos
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.
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/PMC6962699/
https://www.ncbi.nlm.nih.gov/pubmed/31970203
http://dx.doi.org/10.1016/j.omtm.2019.11.023

Internet

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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