Investigator evaluated the Nec-1 in R6/2 transgenic mouse model of HD, which expresses exon 1 of mutant human htt gene.75 Given that Nec-1 can cross the blood-brain barrier easily but has a short half-life, about 1 h,76 Zhu et al. delivered Nec-1 intracerebroventricularly with Alzet osmotic pump to ensure continuous supply of the drug.44 Consequently, the expression of full-length RIP1 protein is increased in R6/2 mice compared with wild-type control.44 Nec-1 treatment helped maintain the motor functions and body weights in the R6/2 mice and significantly delayed disease onset. The gene discussed is PCSK1; the disease is Huntington disease.