Article

A single-nucleus transcriptomic atlas of human basal ganglia during development forwarding diagnosis and therapy of pediatric movement disorders

2026-06-04

Abstract excerpt

Gene therapy is rapidly emerging as a transformative treatment for monogenic neurological disorders, including pediatric movement disorders such as aromatic L-amino acid decarboxylase (AADC) deficiency. However, its success critically depends on defining target cells and windows for therapeutic intervention. Here, we present an open-access single-nucleus transcriptomic atlas of the human basal ganglia spanning a t...

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Literature Corpus work: 194d720b-9d81-52e2-9ac0-b1706d847b1c
DOI: 10.64898/2026.06.04.26354648

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