Rational ASO Design in CNS Disorders: Cell-Type Targeting & Functional Validation in Complex BBBoid In Vitro Systems
Time: 8:30 am
day: Day 2
Details:
- Design antisense oligonucleotides against genetically defined mutations in diseases like Alzheimer’s, ALS, and autism offers a powerful advantage when using in vitro models that accurately reflect the human genetic background
- Discuss the importance of assessing the functional consequences of target modulation, leveraging multiplexed molecular and phenotypic readouts to understand downstream effects in disease-relevant pathways
- Iterative optimization of ASO chemistry, dosing, and delivery enables enhanced cell-type specificity, which is critical when working without access to human in vivo systems
- Develop and tailor platform technologies that can adapt to disease-specific needs is key to building biomimetic in vitro systems with translational relevance across neurodegenerative and neuropsychiatric disorders
