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Discovery of a novel TFEB activator targeting lysosomal dysfunction in amyotrophic lateral sclerosis using artificial intelligence-based virtual screening - PubMed

7 hours ago
  • #TFEB activator
  • #AI screening
  • #neurodegenerative diseases
  • Lysosomal dysfunction is a key feature in neurodegenerative diseases like ALS.
  • TFEB is a target for restoring lysosomal health; AI screening identified isoginkgetin (ISO) as a potent TFEB activator.
  • ISO promotes lysosomal biogenesis, enhances function, and shows neuroprotective effects in ALS models.
  • The study validates AI-driven screening as a paradigm for discovering therapies against ALS and similar diseases.