In the same mouse-brain dataset, GPT-Rosalind found a possible clue to why TSC2 mutations tend to cause more severe tuberous sclerosis than TSC1 mutations. The two genes work together to regulate mTOR. Yet in one group of cerebellar neurons, disrupting TSC2 changed the expression of dozens of genes, including genes involved in neuronal communication. Disrupting TSC1 had little effect on gene expression in the same cell type. Existing treatments already target mTOR. Do they reverse these changes, or is there more to TSC2’s role in these neurons?

GPT-Rosalind surfaced a new lead for drug discovery: could restoring cholesterol transport protect neurons from degeneration? DCTN1 is a gene linked to Perry syndrome, a rare neurodegenerative disorder. In mouse-brain data, disrupting it increased expression of genes involved in making cholesterol. GPT-Rosalind suggested a possible explanation: cholesterol may not be reaching where neurons need it. That gives scientists a specific mechanism to test and, if confirmed, a direction for developing new treatments for neurodegenerative disorders. With @perturbai_tx and @scrippsresearch, a team of thousands of GPT-Rosalind agents worked in parallel, connecting gene-expression results from millions of mouse brain cells with scientific literature in just 32 minutes. We’re now expanding this collaboration to identify new drug targets. openai.com/index/scaling-ai-…