Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain

GM2 gangliosidoses, including Tay–Sachs disease and Sandhoff disease, are lysosomal storage disorders caused by deficiencies in β-N-acetylhexosaminidase (Hex). Patients are afflicted primarily with progressive central nervous system (CNS) dysfunction. Studies in mice, cats, and sheep have indicated...

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Detalhes bibliográficos
Publicado no:Hum Gene Ther
Main Authors: Golebiowski, Diane, van der Bom, Imramsjah M.J., Kwon, Churl-Su, Miller, Andrew D., Petrosky, Keiko, Bradbury, Allison M., Maitland, Stacy, Kühn, Anna Luisa, Bishop, Nina, Curran, Elizabeth, Silva, Nilsa, GuhaSarkar, Dwijit, Westmoreland, Susan V., Martin, Douglas R., Gounis, Matthew J., Asaad, Wael F., Sena-Esteves, Miguel
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2017
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Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5488349/
https://ncbi.nlm.nih.gov/pubmed/28132521
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2016.109
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