Therapeutic Potential of Intracerebroventricular Replacement of Modified Human β-Hexosaminidase B for GM2 Gangliosidosis

To develop a novel enzyme replacement therapy for neurodegenerative Tay-Sachs disease (TSD) and Sandhoff disease (SD), which are caused by deficiency of β-hexosaminidase (Hex) A, we designed a genetically engineered HEXB encoding the chimeric human β-subunit containing partial amino acid sequence of...

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Detalles Bibliográficos
Autores principales: Matsuoka, Kazuhiko, Tamura, Tomomi, Tsuji, Daisuke, Dohzono, Yukie, Kitakaze, Keisuke, Ohno, Kazuki, Saito, Seiji, Sakuraba, Hitoshi, Itoh, Kohji
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2011
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3129794/
https://www.ncbi.nlm.nih.gov/pubmed/21487393
http://dx.doi.org/10.1038/mt.2011.27

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