Targeting neuronal lysosomal dysfunction caused by β-glucocerebrosidase deficiency with an enzyme-based brain shuttle construct

Mutations in glucocerebrosidase cause the lysosomal storage disorder Gaucher’s disease and are the most common risk factor for Parkinson’s disease. Therapies to restore the enzyme’s function in the brain hold great promise for treating the neurological implications. Thus, we developed blood-brain ba...

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Detalles Bibliográficos
Autores principales: Gehrlein, Alexandra, Udayar, Vinod, Anastasi, Nadia, Morella, Martino L., Ruf, Iris, Brugger, Doris, von der Mark, Sophia, Thoma, Ralf, Rufer, Arne, Heer, Dominik, Pfahler, Nina, Jochner, Anton, Niewoehner, Jens, Wolf, Luise, Fueth, Matthias, Ebeling, Martin, Villaseñor, Roberto, Zhu, Yanping, Deen, Matthew C., Shan, Xiaoyang, Ehsaei, Zahra, Taylor, Verdon, Sidransky, Ellen, Vocadlo, David J., Freskgård, Per-Ola, Jagasia, Ravi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10097658/
https://www.ncbi.nlm.nih.gov/pubmed/37045813
http://dx.doi.org/10.1038/s41467-023-37632-4

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