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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Dettagli Bibliografici
Pubblicato in:Mol Genet Metab Rep
Autori principali: 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.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Elsevier 2021
Soggetti:
Research Paper
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC8025141/
https://ncbi.nlm.nih.gov/pubmed/33854948
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2021.100748
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