Retinal gene therapy

AbstractIntroductionInherited retinal diseases are the leading cause of sight impairment in people of working age in England and Wales, and the second commonest in childhood. Gene therapy offers the potential for benefit.Sources of dataPubmed and clinicaltrials.gov.Areas of agreementGene therapy can improve vision inRPE65-associated Leber Congenital Amaurosis (RPE65-LCA). Potential benefit depends on efficient gene transfer and is limited by the extent of retinal degeneration.Areas of controversyThe magnitude of vision improvement fromRPE65-LCA gene therapy is suboptimal, and its durability may be limited by progressive retinal degeneration.Growing pointsThe safety and potential benefit of gene therapy for inherited and acquired retinal diseases is being explored in a rapidly expanding number of trials.Areas timely for developing researchDevelopments in vector design and delivery will enable greater efficiency and safety of gene transfer. Optimization of trial design will accelerate reliable assessment of outcomes.
Source: British Medical Bulletin - Category: General Medicine Source Type: research