Gene targeting as a therapeutic avenue in diseases mediated by the complement alternative pathway.

ABSTRACT: The complement alternative pathway (AP) is implicated in numerous diseases affecting many organs, ranging from the rare hematological disease paroxysmal nocturnal hemoglobinuria (PNH), to the common blinding disease age-related macular degeneration (AMD). Critically, the AP amplifies any activating trigger driving a downstream inflammatory response; thus, components of the pathway have become targets for drugs of varying modality. Recent validation from clinical trials using drug modalities such as inhibitory antibodies has paved the path for gene targeting of the AP or downstream effectors. Gene targeting in the complement field currently focuses on supplementation or suppression of complement regulators in AMD and PNH, largely because the eye and liver are highly amenable to drug deliver

SUBMITTER: Dreismann AK 

PROVIDER: S-EPMC10099504 | biostudies-literature | 2023 Jan

REPOSITORIES: biostudies-literature