Sustained normalization of neurological disease after intracranial gene therapy in a feline model**

Progressive debilitating neurological defects characterize feline GM1 gangliosidosis, a lysosomal storage disease caused by deficiency of lysosomal β-galactosidase. No effective therapy exists for affected children, who often die before age 5. In the current study, an adeno-associated viral vector c...

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Detalhes bibliográficos
Publicado no:Sci Transl Med
Main Authors: McCurdy, Victoria J., Johnson, Aime K., Gray-Edwards, Heather, Randle, Ashley N., Brunson, Brandon L., Morrison, Nancy E., Salibi, Nouha, Johnson, Jacob A., Hwang, Misako, Beyers, Ronald J., Leroy, Stanley G., Maitland, Stacy, Denney, Thomas S., Cox, Nancy R., Baker, Henry J., Sena-Esteves, Miguel, Martin, Douglas R.
Formato: Artigo
Idioma:Inglês
Publicado em: 2014
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4412602/
https://ncbi.nlm.nih.gov/pubmed/24718858
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/scitranslmed.3007733
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