In-vivo gene therapy for Retinitis Pigmentosa

视网膜色素变性是世界范围内导致失明的主要原因之一。RP是一种高度异质性的疾病，由60多个基因中的一个突变引起。由于该病的遗传性，体内基因治疗被认为是治疗RP的最有前途的方法。然而，开发RP的基因治疗的挑战是许多潜在的基因突变是极其罕见的，使得它们对治疗开发没有吸引力。一种允许开发一种单一的精确治疗方法的方法是非常可取的，该方法将使更多的患者群体受益，而不考虑潜在的遗传原因。拟议的项目将为RP的基因不可知疗法开发一种概念证明，这将使更广泛的患者群体受益。

Retinitis Pigmentosa (RP) is one of the leading causes of blindness worldwide. RP is a highly heterogeneous disease caused by mutations in one of over 60 genes. Due to the genetic nature of the disease, in-vivo gene therapy is considered to be the most promising approach to treating RP.However, the challenge of developing gene therapies for RP is that many of the underlying gene mutations are ultra-rare making them unattractive for therapeutic development. An approach that allows the development of a single precision therapy that would benefit a larger patient population irrespective of the underlying genetic cause is highly desirable. The proposed project will develop a proof of concept for a gene agnostic therapy for RP that will benefit a broader patient population.