Author

Rebecca J. Holley

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Open access Sep 2026

Clinical and biochemical correction of a patient with neurodegenerative Mucopolysaccharidosis IIIA using hematopoietic stem cell gene therapy.

Autologous haematopoietic stem cell gene therapy (HSC-GT) is an emerging therapy for treatment-refractory neurodegenerative lysosomal storage disorders (LSD). Mucopolysaccharidosis IIIA (MPSIIIA) is a devastating LSD where pathogenic variants in the SGSH gene lead to toxic heparan sulfate accumulation, inflammation and...

J. Kinsella, Simon A. Jones, K. Buckland et al. · Molecular Therapy · 0 citations

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