LV.CD11b.NAGLU

**LV.CD11b.NAGLU** is a high-titre lentiviral vector designed for ex vivo hematopoietic stem cell (HSC) gene therapy in mucopolysaccharidosis type IIIB (MPS IIIB), a rare neurodegenerative lysosomal storage disorder caused by NAGLU deficiency. It drives myeloid-specific expression of a codon-optimized human **α-N-acetylglucosaminidase (NAGLU)** transgene under the **CD11b promoter**, enabling transduced HSCs to produce and secrete functional NAGLU enzyme after transplantation into myeloid-ablated hosts. Preclinical studies in MPS IIIB mouse models demonstrate robust NAGLU overexpression (up to 13% of wild-type levels in brain), normalization of heparan sulfate storage, reduction in neuroinflammation (e.g., cytokines MIP-1α, IL-1α, MCP-1), correction of astrocytosis and microgliosis, and behavioral improvements, highlighting its potential to address CNS pathology via macrophage-mediated cross-correction.[2][4]