The article discusses the safety and efficacy of EDIT-101, a CRISPR-Cas9 gene-editing therapy, in treating *CEP290*-associated inherited retinal degeneration. The study was a phase 1–2, open-label, single-ascending-dose trial involving 14 participants aged 3 years and older, including 12 adults and 2 children. The primary outcome was safety, and key secondary outcomes included changes in visual acuity, retinal sensitivity, mobility test scores, and vision-related quality of life. No serious adverse events related to the treatment were recorded, and most treatment-related ocular adverse events were mild or moderate. Six participants showed meaningful improvement in cone-mediated vision, and nine participants had meaningful improvements in visual acuity, retinal sensitivity, or mobility test scores. The results support further research into CRISPR-Cas9 gene editing for treating inherited retinal degenerations caused by the *CEP290* IVS26 variant.The article discusses the safety and efficacy of EDIT-101, a CRISPR-Cas9 gene-editing therapy, in treating *CEP290*-associated inherited retinal degeneration. The study was a phase 1–2, open-label, single-ascending-dose trial involving 14 participants aged 3 years and older, including 12 adults and 2 children. The primary outcome was safety, and key secondary outcomes included changes in visual acuity, retinal sensitivity, mobility test scores, and vision-related quality of life. No serious adverse events related to the treatment were recorded, and most treatment-related ocular adverse events were mild or moderate. Six participants showed meaningful improvement in cone-mediated vision, and nine participants had meaningful improvements in visual acuity, retinal sensitivity, or mobility test scores. The results support further research into CRISPR-Cas9 gene editing for treating inherited retinal degenerations caused by the *CEP290* IVS26 variant.