Grant Award Details

A Novel Therapy for Sanfilippo B
Grant Type: 
Grant Number: 
DISC2-14047
Project Objective: 
  • Hypoimmune, gene-modified ESC-derived NPC product that overexpresses NAGLU to treat San Filippo B
Investigator: 
Disease Focus: 
Metabolic Disorders
Neurological Disorders
Sanfilippo Syndrome/MPS III
Human Stem Cell Use: 
Embryonic Stem Cell
Award Value: 
$2,297,884
Status: 
Pre-Active

Grant Application Details

Application Title: 
  • A Novel Therapy for Sanfilippo B
Public Abstract: 

Research Objective

To develop a Stem Cell therapy for Sanfilippo B syndrome.

Impact

There is no treatment for Sanfilippo syndrome, and other therapeutic approaches have failed in clinics. This proposal will develop a stem cell based therapy for Sanfilippo syndrome.

Major Proposed Activities

  • Generation of universal donor Embryonic Stem Cells (ESC H1) using state of the art genome editing technique.
  • Increase the level of the missing enzyme in universal donor ESC (H1) using state of the art genome editing technique.
  • Differentiate ESC into Brain stem cells in vitro capable of secreting NAGLU (NAGLU-NPC).
  • Transplantation of NAGLU-NPC to evaluate if the cells can survive in the mouse brain and can repair brain tissue provide NAGLU enzyme.
  • Transplantation of NAGLU-NPC to evaluate if cells differentiate into functional neuron and integrate in the neuronal networks.
  • Transplantation of NAGLU-NPC to evaluate if cells can repair brain tissue and correct abnormal mouse behavior associated with Sanfilippo syndrome.
Statement of Benefit to California: 

This application will help develop a stem cell therapy for Sanfilippo B disorder, a pediatric genetic disorder that currently has no treatment. If successful, this approach could be extended to several other lysosomal storage diseases, bringing a therapy for these catastrophic disorders.

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