... the FTLD-associated gene TER94 (an ortholog of VCP /p97) and found that the knockdown (KD) of this gene caused premature lethality, reduction in brain volume and alterations in the morphology of mushroom bodies. The changes caused by TER94 KD were rescued by wild-type TER94 but not by the human disease...

... and/or pharmacological therapies. To generate VCP disease models, we used CRISPR/Cas9 gene editing to introduce VCP mutations at the endogenous Drosophila Ter94/dVCP locus, hereafter referred to as VCP ( Fig. 1 A). The VCP gene includes six exons, and all ten pathogenic mutations selected in this study ( Fig...