Targeting Autophagy in Sanfilippo Syndrome

08 Mar 2020

The Sanfilippo Children’s Foundation, together with our Swiss friends Fondation Sanfilippo Suisse, have awarded funds to Dr. Louise O’Keefe from The University of Adelaide and the South Australian Health and Medical Research Institute (SAHMRI).

The one-year project will use Sanfilippo Syndrome fruit fly models to study autophagy, a natural process inside cells, which helps to remove and recycle cell components that are not required or no longer working.

The project will investigate whether increasing autophagy levels can improve symptoms of Sanfilippo, and which parts of this process could be targeted for the development of future therapeutics.

Megan Donnell, Executive Director of the Sanfilippo Children’s Foundation, said: “There is currently significant research being undertaken into autophagy as a therapeutic target in a number of neurodegenerative diseases, including Alzheimer’s disease and Parkinson’s disease. The results of this study will help to identify how autophagy could be targeted to develop therapies for children with Sanfilippo.”

Read more on the project here