1. Abeliovich, Asa, Hefti, Franz, Sevigny, Jeffrey. . Gene Therapy for Parkinson's Disease Associated with GBA1 Mutations. In Journal of Parkinson's disease, 11, S183-S188. doi:10.3233/JPD-212739. https://pubmed.ncbi.nlm.nih.gov/34151863/
2. Granek, Zuzanna, Barczuk, Julia, Siwecka, Natalia, Kucharska, Ewa, Majsterek, Ireneusz. 2023. GBA1 Gene Mutations in α-Synucleinopathies-Molecular Mechanisms Underlying Pathology and Their Clinical Significance. In International journal of molecular sciences, 24, . doi:10.3390/ijms24032044. https://pubmed.ncbi.nlm.nih.gov/36768367/
3. Wang, Chung-Hsing, Huang, Yu-Nan, Liao, Wen-Ling, Su, Pen-Hua, Tsai, Fuu-Jen. 2024. GBA1 as a risk gene for osteoporosis in the specific populations and its role in the development of Gaucher disease. In Orphanet journal of rare diseases, 19, 144. doi:10.1186/s13023-024-03132-x. https://pubmed.ncbi.nlm.nih.gov/38575988/
4. Stirnemann, Jérôme, Belmatoug, Nadia, Camou, Fabrice, Billette de Villemeur, Thierry, Berger, Marc G. 2017. A Review of Gaucher Disease Pathophysiology, Clinical Presentation and Treatments. In International journal of molecular sciences, 18, . doi:10.3390/ijms18020441. https://pubmed.ncbi.nlm.nih.gov/28218669/
5. Huang, Qin, Chan, Ken Y, Lou, Shan, Chan, Yujia A, Deverman, Benjamin E. 2023. An AAV capsid reprogrammed to bind human Transferrin Receptor mediates brain-wide gene delivery. In bioRxiv : the preprint server for biology, , . doi:10.1101/2023.12.20.572615. https://pubmed.ncbi.nlm.nih.gov/38187643/
6. Huang, Qin, Chan, Ken Y, Wu, Jason, Chan, Yujia A, Deverman, Benjamin E. 2024. An AAV capsid reprogrammed to bind human transferrin receptor mediates brain-wide gene delivery. In Science (New York, N.Y.), 384, 1220-1227. doi:10.1126/science.adm8386. https://pubmed.ncbi.nlm.nih.gov/38753766/
7. den Heijer, Jonas M, Kruithof, Annelieke C, Moerland, Matthijs, Groeneveld, Geert Jan, Lansbury, Peter. 2023. A Phase 1B Trial in GBA1-Associated Parkinson's Disease of BIA-28-6156, a Glucocerebrosidase Activator. In Movement disorders : official journal of the Movement Disorder Society, 38, 1197-1208. doi:10.1002/mds.29346. https://pubmed.ncbi.nlm.nih.gov/37195859/
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