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Our Projects

Optimising gene therapy for Sanfilippo Syndrome Type B

 

  • Project title: Gene therapy with intracranial delivery of capsid enhanced AAV NAGLU (MPSIII B)
  • Chief investigator: Dr. Coy Heldermon 
  • Amount: $90K (over 1 year)
  • Location: University of Florida, USA
  • Status: Active  
  • Start date: March 2017

 

Read more about Dr. Heldermon's research here

 

Investigating potential of stem cell therapy for Sanfilippo Type B

 

  • Project title: Neural and Mesenchymal stem cell mediated gene therapy for MPS IIIB
  • Chief investigator: Dr. Coy Heldermon
  • Amount: $90K (over 1 year)
  • Location: University of Florida, USA
  • Start date: March 2017

 

Read more about Dr. Heldermon's research here 

 

Improving delivery of gene therapy to brain for Sanfilippo Type C

 

  • Project title: Improving brain delivery of adeno-associated gene therapy vectors for the treatment of MPS IIIC and other neurodegenerative diseases 
  • Chief Investigator: Dr. Brian Bigger
  • Amount: $90K (1 year)
  • Location: University of Manchester, UK
  • Start date: March 2017 

 

 Read more about Dr. Brian Bigger's research here

 

High throughput screening for new drugs to treat Sanfilippo

  • Project title: New therapy for six Mucopolysaccharidoses
  •  Chief Investigator: Associate Professor Dr Andreas Schulze
  •  Amount: $448K (over 2 years)
  •  Location: Hospital for Sick Children, Toronto, Canada
  •  Start Date: March 2017

 

Read more about Assoc. Prof. Dr. Andreas Schulze's research here

 

Investigating the role of brain lipids (fats) in Sanfilippo

  • Project title: Brain lipid homeostasis: A critical event in Sanfilippo A neuropathology
  • Chief Investigator: Associate Professor Maria Fuller
  • Amount: $90,900 (1 year)
  • Location: SA Pathology, Adelaide
  • Grant Type: Incubator
  • Status: Active  

 

Read more about Assoc. Prof. Maria Fuller's research here 

 

Abeona Therapeutics Gene Therapy Program

 

  • Project title:  ABO-102 Gene Therapy for Patients with Sanfilippo Syndrome Type A (MPS IIIA) and  ABO-101 Gene Therapy for Patients with Sanfilippo Type B (MPS IIIB)
  • Chief Investigator: Abeona Therapeutics
  • Amount $USD 1 million
  • Status: Active

 

Read more about Abeona's clinical trial here

 

PhD Scholarship - SAHMRI

 

  • Project Title: How are the neurons damaged in Sanfilippo and how can it be prevented? 
  • Chief Investigator: Andrew Schoubridge University of Adelaide PhD project will be carried out under the guidance of Dr Kim Hemsley at the Lysosomal Diseases Research Unit at the South Australian Health and Medical Research Institute (SAHMRI).
  • Amount: $15,000 over 3 years
  • Status: Active 

 

Read more about Andrew Schoubridge's research here 

 

Read further about our Research Strategy our Research Funding Program and our Scientific Advisory Board