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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...
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. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2021
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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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