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Sponsored AI AM CLAUDE, A Beginner's Guide: Learn the basics of working with AI AI AM CLAUDE, A Beginner's Guide: Learn the basics of working with AI

Author

Amanda L. Gross

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Open access Sep 2026

Expanded Access use of Intravenous Gene Transfer with AAV9-GLB1 in a Juvenile GM1 Gangliosidosis Participant

GM1 gangliosidosis is an inherited, progressively neurodegenerative lysosomal storage disorder with no approved therapy. We report 5-year safety and 3-year clinical, biochemical, and neuroimaging efficacy outcomes following expanded-access intravenous AAV9-GLB1 gene therapy in GT01, a 6-10-year-old female with juvenile...

Connor J. Lewis, Hera Akmal, Precilla D'Souza et al. · 0 citations
Open access Sep 2026

Single-cell multiomics across nine mammals reveals cell-type-specific regulatory conservation in the brain.

Understanding gene regulation in the brain is essential for defining the genetic basis of neurologic disease. Cis-regulatory elements (CREs) regulate gene expression, but their function and evolution depend on sequence and cis- and trans-regulatory context. We generated single-nucleus RNA- and ATAC-seq data from cortex...

Ashlyn G. Anderson, B. Rogers, Erin A. Barinaga et al. · 0 citations

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