Development and Testing of Sanfilippo Types A, B
and C Zebrafish Models

The Sanfilippo Children’s Foundation has awarded funds to Associate Professor Michael Lardelli at The University of Adelaide, who will develop three zebrafish models of Sanfilippo and investigate potential therapeutic avenues.

Sanfilippo Syndrome is a form of childhood dementia that currently has no approved treatment or cure. To better understand Sanfilippo and identify potential therapeutic avenues, researchers can use model organisms like zebrafish to mimic the disease found in humans. 

With an Incubator Grant awarded by the Sanfilippo Children’s Foundation, along with a grant from the Carthew Family Charity Trust, Assoc. Prof. Lardelli will create three new Sanfilippo zebrafish models: one for each of the subtypes A, B and C. Once the zebrafish are developed, they will undergo behavioural testing to examine their cognition. 

Then, through the use of powerful genetic techniques, Assoc. Prof. Lardelli and his team will analyse which other genes are turned on and off in the brains of the Sanfilippo zebrafish. This genetic analysis may provide clues on disease mechanisms or new pathways for therapeutic intervention. In the future, the zebrafish could also be used to test emerging Sanfilippo therapies on the road towards a clinical trial.

Assoc. Prof. Lardelli has significant experience using zebrafish to study an inherited early-onset form of Alzheimer’s disease, which occurs before the age of 65. Using these Alzheimer’s zebrafish models, he has found that disrupted iron absorption may contribute to neurodegeneration. Now, he will translate knowledge gained from his Alzheimer’s studies into the Sanfilippo realm, including whether iron absorption may also contribute to Sanfilippo symptoms.

Victoria Bowring, CEO of the Sanfilippo Children’s Foundation, said: “These new zebrafish will serve as additional tools for researchers to study Sanfilippo and explore potential treatments. We are very excited for Michael to join the Sanfilippo research community and apply his expertise in Alzheimer’s to further Sanfilippo research.”

“Through a collaborative, united effort between these fields, we hope to develop a greater understanding of the dementias as a whole, which can be used to identify new therapies,” added Founder and Director of the foundation, Megan Donnell.

Assoc. Prof. Michael Lardelli has over 30 years of experience as a geneticist, with the last 18 of those involved in Alzheimer’s Disease research. His laboratory at the University of Adelaide is the world’s leading laboratory using zebrafish to investigate Alzheimer’s Disease. Throughout his career, he has used model organisms and cutting edge genetic techniques to answer key questions about human diseases. 


  • Project title: Intra-family analysis of behaviour and brain transcriptomes of MPS IIIA, B, & C model zebrafish
  • Chief investigator: Assoc. Prof. Michael Lardelli 
  • Amount: $90,000
  • Duration: 1 year 
  • Location: The University of Adelaide
  • Status: Active
  • Start date: January 2021

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