Scientific Research Grants

Mesenchymal Stem Cells for Sanfilippo Syndrome

Engineering Mesenchymal Stem Cells (MSCS) to Produce Sulfamidase for Intrathecal Treatment of Sanfilippo SyndromeAwardee: Jan Nolta, PhD, Director of UC Davis Stem Cell Program, University of California Davis, Institute for Regenerative CuresStart Date: August 2017

New Therapy for Six Mucopolysaccharidoses

Cure Sanfilippo Foundation is co-funding this project in Collaboration with Sanfilippo Children’s Foundation (Australia).Project Summary: Substrate reduction therapy by targeting the N-deacetylase/N-sulfotransferase (NDST) isozymes, NDST1, represents a promising...

Research Strategy for Pre-Existing AAV Antibodies

Cure Sanfilippo Foundation has awarded a grant to Haiyan Fu, PhD, at Nationwide Children's Hospital in Columbus, OH, to research strategies for overcoming a patient's pre-existing AAV (Adeno-Associated Virus) antibodies so gene therapy using AAV is accessible to...

Re-Purposing of FDA-Approved Drugs for LSD

Re-Purposing of FDA-Approved Drugs for the Treatment of LYSOSOMAL STORAGE DISEASES (LSD)Project is funded by Cure Sanfilippo FoundationProject Summary: The study aims to identify FDA-approved compounds that activate the clearance of pathologic lysosomal accumulation...

Research Extension: Intranasal Delivery of Sulfamidase

Cure Sanfilippo Foundation is funding an extension of research by Jeff Esko, PhD, Co-Director Glycobiology Research & Training Center of  University of California San Diego School of Medicine, to explore intranasal delivery of sulfamidase as a therapy for...

Synaptic Dysfunction in Sanfilippo Type C

Cure Sanfilippo Foundation has awarded a grant to Alexey Pshezhetsky, PhD, of St. Justine University Hospital (Montreal, CA) to explore the synaptic dysfunctions in Sanfilippo Syndrome Type C.Project Summary: The study’s major objective is to understand the...

Testing Compounds on MPS III Fibroblasts

Cure Sanfilippo Foundation has awarded a grant to Alexey Pshezhetsky, PhD, of St. Justine University Hospital (Montreal, CA) to conduct compound testing on MPS III fibroblasts.Project Summary: Results of two previously-published studies in which MPS IIIA fibroblasts...

Intranasal Delivery of Sulfamidase for Sanfilippo

Cure Sanfilippo Foundation is funding research by Jeff Esko, PhD, Co-Director Glycobiology Research & Training Center of University of California San Diego School of Medicine, to explore intranasal delivery of sulfamidase as a therapy for Sanfilippo...