Cure Sanfilippo Foundation is co-funding this gene therapy-approach project with Sanfilippo Children’s Foundation (Australia).
Project Summary: Current therapy for Sanfilippo Syndrome (MPS III) is aimed at restoring functional enzyme or reducing heparan sulfate storage, but does not address restoration of the damage done prior to therapy. Regeneration of damaged central nervous system and prevention or reversal of immune activation by neural stem cells (NSC) and mesenchymal stem cells (MSC) may be necessary in the treatment of MPSIIIB.
This study will evaluate the effect of transplanting NSC and MSC on immune system modulation and regenerative repair. Using lentiviralvector gene-modified stem cells to over express N-acetylglucosaminidase (NAGLU), the study will evaluate the response and therapeutic benefit of each cell type on MPSIIIB mice and controls.
This strategy uses a cell-based, gene-therapy approach to address two priority areas:
- Halt disease progression (Enzyme replacement, gene therapy, cell therapy to stop progression) and
- Repair Damage (Repair and reverse cell damage).