Table 5.

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

• 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.