Protease-resistant modified human β-hexosaminidase B ameliorates symptoms in GM2 gangliosidosis model

GM2 gangliosidoses, including Tay-Sachs and Sandhoff diseases, are neurodegenerative lysosomal storage diseases that are caused by deficiency of β-hexosaminidase A, which comprises an αβ heterodimer. There are no effective treatments for these diseases; however, various strategies aimed at restoring...

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Publicado en:J Clin Invest
Main Authors: Kitakaze, Keisuke, Mizutani, Yasumichi, Sugiyama, Eiji, Tasaki, Chikako, Tsuji, Daisuke, Maita, Nobuo, Hirokawa, Takatsugu, Asanuma, Daisuke, Kamiya, Mako, Sato, Kohei, Setou, Mitsutoshi, Urano, Yasuteru, Togawa, Tadayasu, Otaka, Akira, Sakuraba, Hitoshi, Itoh, Kohji
Formato: Artigo
Idioma:Inglês
Publicado: American Society for Clinical Investigation 2016
Assuntos:
Research Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC4855921/
https://ncbi.nlm.nih.gov/pubmed/27018595
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI85300
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