PGRN deficiency exacerbates, whereas a brain penetrant PGRN derivative protects, GBA1 mutation-associated pathologies and diseases

Mutations in GBA1, encoding glucocerebrosidase (GCase), cause Gaucher disease (GD) and are also genetic risks in developing Parkinson’s disease (PD). Currently, the approved therapies are only effective for directly treating visceral symptoms, but not for primary neuronopathic involvement in GD (nGD...

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Detalles Bibliográficos
Autores principales: Zhao, Xiangli, Lin, Yi, Liou, Benjamin, Fu, Wenyu, Jian, Jinlong, Fannin, Venette, Zhang, Wujuan, Setchell, Kenneth D. R., Grabowski, Gregory A., Sun, Ying, Liu, Chuan-ju
Formato: Online Artículo Texto
Lenguaje:English
Publicado: National Academy of Sciences 2022
Materias:
Biological Sciences
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9910439/
https://www.ncbi.nlm.nih.gov/pubmed/36574647
http://dx.doi.org/10.1073/pnas.2210442120

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9910439/
https://www.ncbi.nlm.nih.gov/pubmed/36574647
http://dx.doi.org/10.1073/pnas.2210442120

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