Reduced heparan sulfate levels in cerebrospinal fluid reflect brain neuron correction in Sanfilippo B mice.

Le, Steven Q; Sorensen, Alexander; Sukupolvi, Soila; Jewhurst, Gianna; Austin, Grant; Doray, Balraj; Cooper, Jonathan D; Dickson, Patricia I · J Clin Invest · 2025

basic_science · Level V

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Abstract

A biomarker in spinal fluid reflects brain disease in a rare genetic disorder, with implications for how we diagnose and monitor the disease.

Medical subject headings