Our Projects
Therapeutic strategies for the treatment of behavioural symptoms
- Project title: New Therapeutic strategies for the treatment of behavioural symptoms in Sanfilippo
- Chief investigator: Dr. Elvira De Leonibus
- Amount: $45,000 with Cure Sanfilippo Foundation contributing a further $45,000
- Duration: 1 year
- Location: Telethon Institute of Genetics and Medicine (TIGEM), Italy
- Status: Active
- Start date: March 2019
*Sanfilippo Type A will be studied in this project but the findings could be applied to all types of Sanfilippo
Read more about Dr. Leonibus' research here
Targeting autophagy as a therapeutic strategy for Sanfilippo
- Project title: Pharmacological induction of autophagy as a novel therapeutic strategy for MPS III A
- Chief Investigators: Dr. Ivan Conte and Dr. Nicolina Cristina Sorrentino
- Amount: $90,000
- Duration: 1 year
- Location: Telethon Institute of Genetics and Medicine, Italy
- Status: Active
- Start date: March 2019
*Sanfilippo Type A will be studied in this project but the findings could be applied to all types of Sanfilippo
Read more about Dr. Conte and Dr. Sorrentino's research here
Searching for "super active" enzymes for Sanfilippo Type A
- Project title: Generation of “super active” variants of lysosomal enzymes to treat the CNS in Sanfilippo Syndrome
- Chief Investigator: Dr Alessandro Fraldi
- Amount: $90, 000
- Duration: 1 year
- Location: Telethon Institute of Genetics and Medicine (TIGEM), Italy
- Status: Active
- Start date: March 2019
Read more about Dr. Fraldi's research here
Creating a zebrafish model for Sanfilippo Type A
- Project title: Creating a MPSIII zebrafish model and brain specific in vivo lysosome reporter for drug screening and in vivo imaging of disease pathology
- Chief Investigator: Dr. Jan Kaslin
- Amount: $45,000 (over 1 year), with Cure Sanfilippo Foundation contributing a further $45,000
- Location: Victoria, Australia
- Status: Active
- Start date: February 2018
Read more about Dr. Kaslin's research here
Designing "chaperone" drugs for Sanfilippo Type A
Read more about Prof. Vito Ferro's research here
Targeting misfolded proteins for therapy for Sanfilippo Type C
Read more about Prof. Alexey Pshezhetsky's research here
Abeona Phase 1B trial of Gene Therapy program
- Project title: Phase 1B trial of gene therapy (ABO-101 & ABO-102)
- Chief Investigator: Abeona Therapeutics
- Amount: USD $1.5 Million
- Status: Commencement TBA
Read more about Abeona Therapuetics Phase 1B 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 in the Childhood Dementia Research Group, as part of the Hopwood Centre for Neurobiology 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
PhD Scholarship - University of Adelaide
- Project title: Investigating the molecular mechanisms that mediate neuropathology in Sanfilippo Syndrome
- Chief Investigator: Laura Hewson’s PhD project will be carried out under the guidance of Dr Lousie O’Keefe at the University of Adelaide. Laura is co-supervised by Dr Kim Hemsley (SAHMRI) and Prof Robert Richards (University of Adelaide)
- Amount: $10,000 over 2 years
- Status: Active
Read more about Laura Hewson's research here
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: $90,000 (over 1 year)
- Location: University of Florida, USA
- Status: Active
- Start Date: March 2017
Read more about Dr. Heldermon's research here
Investigating potential 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: $90,000 (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: $90,000 (1 year)
- Location: University of Manchester, UK
- Status: Completed
- Start date: March 2017
Project completed. Read a Research Update here and about Dr. Bigger's original research projectt 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: $448,000 (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
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