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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Dades bibliogràfiques
Publicat a:Hum Gene Ther
Autors principals: 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
Format: Artigo
Idioma:Inglês
Publicat: Mary Ann Liebert, Inc. 2017
Matèries:
Research Articles
Accés en línia: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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