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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Autori principali: Matsuoka, Kazuhiko, Tamura, Tomomi, Tsuji, Daisuke, Dohzono, Yukie, Kitakaze, Keisuke, Ohno, Kazuki, Saito, Seiji, Sakuraba, Hitoshi, Itoh, Kohji
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2011
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3129794/
https://ncbi.nlm.nih.gov/pubmed/21487393
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2011.27
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