1.We hypothesize that RNA splicing factor retinitis pigmentosa will be amenable to treatment by AAV-mediated gene therapy, and that understanding the clinical progression rates of PRPF31 retinitis pigmentosa will help with the design of gene therapy clinical trials.
我们假设RNA剪接因子色素性视网膜炎可以通过aav介导的基因治疗进行治疗，了解PRPF31色素性视网膜炎的临床进展速度将有助于设计基因治疗临床试验。