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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

Xuntian Jiang

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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 Aug 2026

Metabolomic, lipidomic, and N-glycomic analyses of a human cell model of Krabbe disease reveal treatable deficits in glycosylation and serine-ceramide metabolism

Stable isotope tracing is used to comprehensively characterize metabolic perturbations in a human oligodendrocellular Krabbe disease model and suggests impaired protein glycosylation as a previously unrecognized pathogenic mechanism in Krabbe disease.

Rodrigo T. Starosta, Hannah N. Saeger, J. ten Hoeve et al. · 0 citations

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