To date, proof‐of‐concept of AONs has been shown in both cell‐based models and animal models for four retinal dystrophy genes: CEP290 (Collin et al., 2012; Dulla et al., 2018; Garanto et al., 2016; Parfitt et al., 2016), CHM (Garanto, van der Velde‐Visser, Cremers, & Collin, 2018), RHO (Murray et al., 2015), and USH2A (Slijkerman et al., 2016), and represents a promising therapy for retinal dystrophies (Collin & Garanto, 2017). The gene discussed is CHM; the disease is inherited retinal dystrophy.