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5 Discussion: perspective and open questions While the identification of RAiNs (Sebastian et al., 2012) and VAPB (Janikiewicz et al., 2018), which likely compromise the biophysical environment required for LLPS and biomolecular condensate nucleation in -cells (Huang et al., 2025) or only delay further decay is essential for de-risking clinical translation and defining the therapeutic window for these agents in late-stage pathologies

The choice between these methods depends on individual preferences and the severity of the deficiency