Author

Oriana Mandolfo

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Open access Aug 2026

Human iPSC-derived neural progenitor gene therapy improves outcomes in a neuropathic lysosomal disease.

A neural stem cell gene therapy that achieves high enzyme delivery and restores cognitive function, likely through integration of healthy neural cells across multiple brain regions is reported, offering a promising therapeutic avenue for MPS IIIA and broader lysosomal storage disorders.

Oriana Mandolfo, Yuko Ishikawa Learmonth, Asma’u Usman et al. · Molecular Therapy · 0 citations