Roundtable Discussion: Into the Thick of It: Deep Brain Penetration & Cell-Specific Precision
- Evaluate strategies to achieve robust oligonucleotide penetration into deep brain regions while maintaining controlled CNS distribution
- Examine approaches to enhance neuron-specific uptake over glial populations, improving cell-type precision and therapeutic impact
- Assess the limitations of current delivery systems, including transferrin receptor-mediated approaches, and discuss emerging shuttle technologies for deeper and more targeted brain access
- Leverage imaging and PK/PD modelling to quantify regional and cell-specific exposure, enabling early differentiation between delivery efficiency and target biology
- Address safety and translational considerations, balancing deep CNS exposure, cell selectivity, and risks of neurotoxicity and peripheral off-target effects