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Examination of a blood-brain barrier targeting β-galactosidase-monoclonal antibody fusion protein in a murine model of GM1-gangliosidosis

GM1-gangliosidosis is a lysosomal disease resulting from a deficiency in the hydrolase β-galactosidase (β-gal) and subsequent accumulation of gangliosides, primarily in neuronal tissue, leading to progressive neurological deterioration and eventually early death. Lysosomal diseases with neurological...

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Detalles Bibliográficos
Autores principales: Przybilla, Michael J., Stewart, Christine, Carlson, Timothy W., Ou, Li, Koniar, Brenda L., Sidhu, Rohini, Kell, Pamela J., Jiang, Xuntian, Jarnes, Jeanine R., O'Sullivan, M. Gerard, Whitley, Chester B.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8025141/
https://www.ncbi.nlm.nih.gov/pubmed/33854948
http://dx.doi.org/10.1016/j.ymgmr.2021.100748

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