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hematopoietic stem cell gene therapy with lentiviral vector encoding hHGSNAT
- Development stage
- Preclinical
- Lead developer
- University of California, San Diego
- Modality
- Gene Therapies, Stem Cell Therapies → Cell Therapies
- Administration
- Intravenous
01
Overview
This is an autologous ex vivo gene therapy for Mucopolysaccharidosis type IIIC (MPSIIIC), a neurodegenerative lysosomal storage disorder. The therapy involves harvesting a patient's hematopoietic stem and progenitor cells (HSPCs) and transducing them with a lentiviral vector encoding the human HGSNAT gene. Once re-infused, these gene-modified cells engraft and differentiate into macrophage/microglia-like cells in various tissues, including the brain. These cells then provide the functional HGSNAT enzyme, which is missing or deficient in MPSIIIC patients, thereby reducing the accumulation of heparan sulfate and ameliorating disease symptoms. This approach is being developed by researchers at the University of California San Diego (UCSD).
- Other names
- gene-modified hematopoietic stem and progenitor cell transplantationautologous ex vivo gene therapy with lentiviral vector encoding hHGSNAT
02
Targets
HGSNAT (Heparan-alpha-glucosaminide N-acetyltransferase)
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