A Novel Therapy for Sanfilippo B
Grant Award Details
Grant Type:
Grant Number:
DISC2-14047
Investigator(s):
Name:
Type:
PI
Disease Focus:
Human Stem Cell Use:
Award Value:
$2,297,884
Status:
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
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.