Intracerebroventricular enzyme replacement therapy with β-galactosidase reverses brain pathologies due to GM1 gangliosidosis in mice

Autosomal recessive mutations in the galactosidase β1 (GLB1) gene cause lysosomal β-gal deficiency, resulting in accumulation of galactose-containing substrates and onset of the progressive and fatal neurodegenerative lysosomal storage disease, GM1 gangliosidosis. Here, an enzyme replacement therapy...

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Bibliografski detalji
Izdano u:J Biol Chem
Glavni autori: Chen, Joseph C., Luu, Amanda R., Wise, Nathan, Angelis, Rolando De, Agrawal, Vishal, Mangini, Linley, Vincelette, Jon, Handyside, Britta, Sterling, Harry, Lo, Melanie J., Wong, Hio, Galicia, Nicole, Pacheco, Glenn, Van Vleet, Jeremy, Giaramita, Alexander, Fong, Sylvia, Roy, Sushmita M., Hague, Chuck, Lawrence, Roger, Bullens, Sherry, Christianson, Terri M., d'Azzo, Alessandra, Crawford, Brett E., Bunting, Stuart, LeBowitz, Jonathan H., Yogalingam, Gouri
Format: Artigo
Jezik:Inglês
Izdano: American Society for Biochemistry and Molecular Biology 2020
Teme:
Cell Biology
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7521651/
https://ncbi.nlm.nih.gov/pubmed/31481471
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1074/jbc.RA119.009811
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