Gliosis-dependent expression of complement factor H truncated variants attenuates retinal neurodegeneration following ischemic injury.

ABSTRACT: Inherited, age-related, and acute retinal diseases are often exacerbated by an aberrant or excessive activity of the complement system. Consequently, cells not directly affected by an acute event or genetic variants may degenerate, resulting in enhanced visual impairment. The therapeutic potential of supplementation of complement factor H (FH), a key regulator of the complement cascade, is therefore particularly promising in the context of retinal diseases caused by complement activation. In this study, we engineered adeno-associated viruses (AAVs) containing sequences of two truncated human FH variants. The expression of these variants was regulated by the glial fibrillary acidic protein (GFAP) promoter, which is selectively active in gliotic Müller cells. Both FH variants consisted of FH

SUBMITTER: Biber J 

PROVIDER: S-EPMC10885619 | biostudies-literature | 2024 Feb

REPOSITORIES: biostudies-literature