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Table 5 Future directions to improve the translation of individual genetic information into novel and personalized treatments for ALS and FTD

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.