Developing novel gene therapy approaches to inhibit the unconventional translation of neurotoxic dipeptide-repeat proteins in pre-clinical models of C

Presented in the form of testable hypotheses.

Design and evaluate the therapeutic potential of our novel neuroprotective strategies using adeno-associated virus serotype 9 (AAV9) gene therapy in C9ORF72-ALS/FTD mice available in the supervisors' groups.

Specifically, we aim to: (i) engineer therapeutic vectors targeting RAN translation-associated factors; (ii) evaluate their efficacy in patient-derived neurons; (iii) test their safety and efficacy in a pilot proof-of-principle study in C9ORF72-ALS/FTD mice.