BDSRA Australia proudly helped fund this work with a $49,400 grant in 2020 and we are thrilled to see the study now published in the peer-review journal ‘Disease Models & Mechanisms’.

Using neurons derived from CLN3 disease patient cells, Cook's team show that neurons expressing mutant (dysfunctional) CLN3 protein have reduced 'cell-to-cell' communication, accumulated cell waste in lysosomes, and showed disruption of several key proteins related to neuron development, axon guidance and lysosome activity.

This human cell model represents an important new tool for CLN3 disease research, through which we can better understand cell changes during CLN3 disease, and for identification of potential new targets for therapy.

Congratulations to Tony, Sueanne and team, and THANK YOU for your tireless work that continues to bring hope to our Batten community!

To read the full article, visit: https://journals.biologists.com/.../Lysosomal-alterations...