Gene Therapy for Dominantly Inherited Retinal Degeneration

摘要: The elucidation of the molecular pathogenesis inherited ocular disorders has led to an ability design therapies directed toward amending primary genetic lesion or modulating secondary effects associated with disease pathologies. Indeed treatment retinal degenerations entered a new era publication seminal gene therapy trials for RPE65-linked Leber congenital amaurosis in 2008. Since then, number encouraging Phase I/II studies have been reported recessively disorders, as many successful preclinical recessive retinopathies typically using augmentation strategy. It is therefore timely discuss promising approaches another category retinopathy, that is, dominantly retinopathies. In this chapter, various are being explored will be discussed. Therapies targeted correcting mutations driving process, course, reviewed. possibility combining such optimize efficacy also