A variety of gene therapy approaches using adeno-associated viral vectors for gene delivery, antisense oligonucleotides or genome editing agents have already yielded promising results to prevent HI and retinal degeneration in murine USH models (Nagel-Wolfrum et al., 2014; Géléoc and El-Amraoui, 2020; Lentz et al. 2020), including clinical trials in patients related to mutations in exon 13 of USH2A (NCT03780257). The gene discussed is USH2A; the disease is retinal degeneration.