Conducting gene therapy clinical trials based on natural history studies for inherited retinal diseases in Chinese population

A breakthrough in the treatments of patients with inherited retinal diseases(IRDs)have been achieved with the development of gene sequencing and gene editing technologies.Luxturna,the world's first IRD gene therapy drug for the treatment of Leber's Congenital Amaurosis(LCA)was launched in 2017.1 It indicates that IRD gene therapy had entered the new stage of clinical application.The new era of gene therapy is coming.However,IRD has a low prevalence and a high degree of genetic and clinical heterogeneity.Most of the existing gene therapy-related researches and drugs were based on the genetic backgrounds of western people.