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Targeting neuronal lysosomal dysfunction caused by β-glucocerebrosidase deficiency with an enzyme-based brain shuttle construct.
Gehrlein A, Udayar V, Anastasi N, Morella ML, Ruf I, Brugger D, von der Mark S, Thoma R, Rufer A, Heer D, Pfahler N, Jochner A, Niewoehner J, Wolf L, Fueth M, Ebeling M, Villaseñor R, Zhu Y, Deen MC, Shan X, Ehsaei Z, Taylor V, Sidransky E, Vocadlo DJ, Freskgård PO, Jagasia R. Gehrlein A, et al. Nat Commun. 2023 Apr 12;14(1):2057. doi: 10.1038/s41467-023-37632-4. Nat Commun. 2023. PMID: 37045813 Free PMC article.
Development of quantitative high-throughput screening assays to identify, validate, and optimize small-molecule stabilizers of misfolded β-glucocerebrosidase with therapeutic potential for Gaucher disease and Parkinson's disease.
Williams D, Glasstetter LM, Jong TT, Kapoor A, Zhu S, Zhu Y, Gehrlein A, Vocadlo DJ, Jagasia R, Marugan JJ, Sidransky E, Henderson MJ, Chen Y. Williams D, et al. Among authors: gehrlein a. bioRxiv [Preprint]. 2024 Mar 27:2024.03.22.586364. doi: 10.1101/2024.03.22.586364. bioRxiv. 2024. PMID: 38712038 Free PMC article. Preprint.