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Development of a targeted BioPROTAC degrader selective for misfolded SOD1 - Nature Communications
Abstract The accumulation of misfolded proteins underlies a broad range of neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS). Due to their dynamic nature, these misfolded proteins have proven challenging to target therapeutically. Here, we specifically target misfolded disease variants of the ALS-associated protein superoxide dismutase 1 (SOD1), using a biological proteolysis targeting chimera (BioPROTAC) composed of a SOD1-specific intrabody and an E3 ubiquitin ligase.
A role for triglyceride lipase brummer in the regulation of sex differences in Drosophila fat storage and breakdown
Loading metrics Open Access Peer-reviewed Research Article Lianna W. Wat, Chien Chao, Rachael Bartlett , Justin L. Buchanan , Jason W. Millington, Hui Ju Chih, Zahid S. Chowdhury, Puja Biswas, Vivian Huang, Leah J. Shin, Lin Chuan Wang, Marie-Pierre L. Gauthier, Maria C. Barone, Kristi L. Montooth, Michael A. Welte, [ ... ], Elizabeth J. Rideout [ view all ] [ view less ] Lianna W. Wat, Chien Chao, Rachael Bartlett, Justin L. Buchanan, Jason W. Millington, Hui Ju Chih, Zahid S.
P2X7 antagonism using Brilliant Blue G reduces body weight loss and prolongs survival in female SOD1G93A amyotrophic lateral sclerosis mice
Conclusions In conclusion, BBG treatment reduced body weight loss and prolonged survival in female SOD1G93A mice. This represents the first report of an extension in survival following P2X7 blockade in an ALS mouse model. Given that this result was seen only in females, the importance of considering sex in pre-clinical studies is clear, consistent with reports by others (Cervetto et al., 2013).
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