Allele-specific knock-down using AONs or RNA interference has already been tested for various targets that exert a gain of function or dominant negative effect, such as TMC1 (MIM 606706) for autosomal dominant hearing loss (MIM 606705) [15], COL6A2 (MIM 120240), and COL6A3 (MIM 120250) for Ullrich myopathy (MIM 254090) [16,17,18], DNM2 (MIM 602378) for autosomal dominant centronuclear myopathy (MIM 160150 [19], RHO (MIM 180380) for adRP [20], GCAP1 (MIM 600364) for adRP, and cone-rod dystrophy (MIM 602093 [21] and HTT (MIM 613004) for Huntington disease (MIM 143100) [22,23]. Here, PLIN2 is linked to juvenile Huntington disease.