In-vivo gene therapy for Retinitis Pigmentosa

色素性视网膜炎的体内基因治疗

• 批准号: 10043370
• 金额: $ 6.37万
• 依托单位: DRISHTI DISCOVERIES LTD.
• 依托单位国家: 英国
• 项目类别: Grant for R&D
• 财政年份: 2022
• 资助国家: 英国
• 起止时间: 2022 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=10043370
• 关键词: vivo; gene; therapy; Retinitis; Pigmentosa

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.

色素性视网膜炎（RP）是世界范围内致盲的主要原因之一。RP是一种高度异质性的疾病，由60多个基因之一的突变引起。由于疾病的遗传性质，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基因治疗的挑战是，许多潜在的基因突变是极其罕见的，使它们对治疗开发没有吸引力。非常需要一种方法，其允许开发将使更大的患者群体受益的单一精确疗法，而不管潜在的遗传原因如何。拟议的项目将为RP的基因不可知疗法开发概念验证，这将使更广泛的患者群体受益。