On Wednesday 7 May 2025, C. Milazzo will defend the doctoral thesis titled: Investigating the Potential of Antisense: Oligonucleotides for the Treatment of Neurodevelopmental Disorders
- Promotor
- Co-promotor
- Date
- Wednesday 7 May 2025, 10:30 - 12:00
- Type
- PhD defence
- Space
- Professor Andries Querido room
- Building
- Education Center
- Location
- Erasmus MC
Below is a brief summary of the dissertation:
Over the past several decades, antisense oligonucleotide (ASO) therapy has been explored as a treatment for various central nervous system (CNS) disorders of genetic origin. Encouraged by the clinical benefits reported in numerous trials and the approval of Nusinersen in 2016 for the treatment of spinal muscular atrophy, our laboratory has investigated the potential of ASOs as a treatment for two neurodevelopmental disorders (NDDs): Angelman Syndrome (AS) and Tuberous Sclerosis Complex (TSC). In this thesis, we explored the efficacy, dose-dependent challenges, treatment duration, and toxicity of ASOs targeting key RNAs implicated in these disorders using mouse models. Our preclinical findings highlight important considerations for the clinical translation of ASO therapies in NDDs and support the potential of ASOs as an effective treatment for patients with AS or TSC.
- More information
The public defence will start exactly at 10.30 hrs. The doors will be closed once the public defence starts, latecomers can access the hall via the fourth floor. Given the solemn nature of the meeting, we advise not to bring children under the age of 6 to the first part of the ceremony.
A livestream link has been provided to candidate.
