Inherited retinal disease (IRD) affects 1 in 2,500 to 3,000 live births, which usually cause severe vision impairment and often blindness.So far over 200 genes have been identified as causative genes of 26 distinct forms of IRDs.Due to the lack of cure, gene therapy for IRDs has been actively pursued during the past two decades.The recent few years have witnessed the success of gene therapy for one form of these diseases in patients.RPE65-LCA clinical trials by four independent groups have shown functional improvements in most recipients.Encouraged by these results, trials for several other forms of IRDs have been approved and are being conducted.The presenter will introduce the treatment strategies, the advancement of AAV vector development and the challenges for IRD gene therapy.The ongoing work of RPGR-retinitis pigmentosa treatment on mouse models will also be presented.