From: From basic research to the clinic: innovative therapies for ALS and FTD in the pipeline
• Gaining novel insights into molecular mechanisms of ALS and FTD pathophysiology by better integration of Clinical, Neuropathological, Neuroimaging, Next-Generation Sequencing, Proteomics, Pharmacogenetics studies. | |
• Characterization of common and divergent mechanisms leading to ALS and FTD. | |
• Revamp of ALS and FTD disease classification system according to the novel genetic and molecular information to identify subgroups of patients that might respond to treatments at a higher (or lower) rate than the population average. | |
• Identification of reliable biomarkers for diagnosing, monitoring the response to therapy, and predicting disease progression. | |
• Development of robust animal models and protocols to minimize eventual off target effects. | |
• Optimization of ASOs' delivery across the blood-brain barrier. | |
• Decrease/bypass the viral vectors' immunogenicity and the eventual pre-existing immunity to AAV. |