The ExonViz tool (https://exonviz.rnatherapy.nl/), which was developed last year, is part of a bigger project called Genetic Therapy Generator Toolkit (Genetic Therapy Generator Toolkit). GTGT automates and accelerates the identification of suitable variants for various genetic treatments. Although the focus of the tool will be on ASOs, it should eventually also be able, among others, to assess variants for gene editing technologies. Feel free to try this tool and let us know what you think.

The genes and diseases with neurological phenotypes that are possibly eligible for ASO therapy have been checked by us and can be found in this list. The list is not exhaustive so please contact us if there is a gene we should add.