TARDBP and frontotemporal dementia: While this proof-of-concept demonstrates the potential for RNA-targeting CRISPR technology to affect TDP-43 proteinopathy, additional work will be needed to answer several important questions beyond those already discussed, including: (i) what is the effectiveness of this approach in other models of ALS-FTD and (ii) can targeting ataxin-2 at a later stage of disease affect or reverse its progression?