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Our goal is to identify a therapy that significantly slows neurodegeneration in individuals with NPC1, and this collaboration will allow us to explore the potential therapeutic efficacy of systemic administration of this novel AAV capsid. Advances in personalized stem cell therapy for Parkinsons disease Scripps Research (CA, USA) presented encouraging interim clinical data at the International Society for Stem Cell Research meeting (ISSCR

Key sensors in the UPRATF6 (activating transcription factor 6), IRE1 (inositol-requiring enzyme 1 alpha), and PERK (protein kinase R-like ER kinase)are activated when misfolded protein concentrations exceed a certain threshold

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These molecules often adopt distinct secondary and tertiary structures, frequently featuring post-translational modifications that are essential for their biological activity (Xuan et al., 2023