PMC · DOI:10.1016/j.ebiom.2026.106129·January 14, 2026
Broadening applications for intrathecal gene therapy: a case for lysosomal storage diseases
eBioMedicine
Abstract
Linked entities
Genes, proteins, chemicals, diseases, species, mutations and cell lines named across the full text — each resolved to its canonical identifier and authoritative record.
Diseases1
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Taxonomy
TopicsNeurogenetic and Muscular Disorders Research · Lysosomal Storage Disorders Research · Hereditary Neurological Disorders