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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Publicado en:Hum Gene Ther
Autores principales: 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
Lenguaje:Inglês
Publicado: Mary Ann Liebert, Inc. 2017
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Research Articles
Acceso en línea: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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