Therapeutic Response in Feline Sandhoff Disease Despite Immunity to Intracranial Gene Therapy

Salutary responses to adeno-associated viral (AAV) gene therapy have been reported in the mouse model of Sandhoff disease (SD), a neurodegenerative lysosomal storage disease caused by deficiency of β-N-acetylhexosaminidase (Hex). While untreated mice reach the humane endpoint by 4.1 months of age, m...

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Detalhes bibliográficos
Main Authors: Bradbury, Allison M, Cochran, J Nicholas, McCurdy, Victoria J, Johnson, Aime K, Brunson, Brandon L, Gray-Edwards, Heather, Leroy, Stanley G, Hwang, Misako, Randle, Ashley N, Jackson, Laura S, Morrison, Nancy E, Baek, Rena C, Seyfried, Thomas N, Cheng, Seng H, Cox, Nancy R, Baker, Henry J, Cachón-González, M Begona, Cox, Timothy M, Sena-Esteves, Miguel, Martin, Douglas R
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2013
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3702100/
https://ncbi.nlm.nih.gov/pubmed/23689599
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.86
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