CRISPR activation of PIKFYVE as potential therapy for FIG4 deficiency
Q Doctrove, Guy M. Lenk, VH LiPuma, MH Meisler
This study uses CRISPR activation to upregulate PIKFYVE in Fig4 deficient mice, showing that a 2-fold increase is insufficient to rescue lysosomal enlargement and survival.
bioRxiv · Apr 28, 2026 · PreprintGenetics