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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Autors principals:	Heather L. Gray-Edwards, Anne S. Maguire, Nouha Salibi, Lauren E. Ellis, Taylor L. Voss, Elise B. Diffie, Jey Koehler, Ashley N. Randle, Amanda R. Taylor, Brandon L. Brunson, Thomas S. Denney, Ronald J. Beyers, Atoska S. Gentry, Amanda L. Gross, Ana R. Batista, Miguel Sena-Esteves, Douglas R. Martin
Format:	Artigo
Idioma:	Inglês
Publicat:	Elsevier 2020-06-01
Col·lecció:	Molecular Therapy: Methods & Clinical Development
Matèries:	gangliosidosis GM1 lysosomal storage disease adeno-associated virus AAV gene therapy
Accés en línia:	http://www.sciencedirect.com/science/article/pii/S2329050119301536
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