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AAV9-Ctsa-Bici-Neu1
- Development stage
- Preclinical
- Lead developer
- UMass Chan Medical School
- Modality
- Gene Therapies
- Administration
- Intrathecal, Intravenous
01
Overview
AAV9-Ctsa-Bici-Neu1 is a preclinical bicistronic adeno-associated virus serotype 9 gene therapy designed to co-deliver functional CTSA and NEU1 genes for the treatment of galactosialidosis and sialidosis. CTSA encodes protective protein cathepsin A, which is required for the stability, lysosomal trafficking, and enzymatic activity of neuraminidase 1 encoded by NEU1. By restoring both components, the vector is intended to reconstitute lysosomal neuraminidase activity, reduce pathological glycoconjugate accumulation, and address central nervous system and peripheral manifestations. UMass Chan Medical School has evaluated combined intracerebroventricular and intravenous administration in Neu1-knockout and Ctsa-knockout mouse models.
02
Targets
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