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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...
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 |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
National Academy of Sciences
2022
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Materias: | |
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 |
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