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截断的补充因子 H Y402 基因疗法拯救了 C3 凝聚氨基炎

Lindsey A Chew1, Daniel Grigsby2, C Garren Hester3

  • 1Department of Ophthalmology, Duke Eye Center, Duke University Medical Center, Durham, NC 27710, USA; Department of Cell Biology, Duke University Medical Center, Durham, NC 27710, USA.

Molecular therapy : the journal of the American Society of Gene Therapy
|April 26, 2025
PubMed
概括

使用腺相关病毒 (AAV) 介导的切断补充因子H (tCFH) 传递的基因疗法对治疗补充介导的疾病如C3球球腺炎 (C3G) 和与年龄相关的黄斑变性 (AMD) 是有前途的. 这种方法恢复了补充通路功能,并在没有免疫排斥的小鼠模型中逆转了疾病.

关键词:
在C3型血球膜炎中.在C3型淋巴细胞病变中.与年龄相关的黄斑变性.补充因子 H 是 H 的补充因子.补充条例补充条例的规定.基因替代是一种基因替代.基因治疗的基因疗法

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