Shared etiology of Mendelian and complex disease supports drug discovery.

ABSTRACT:

Background

Drugs targeting disease causal genes are more likely to succeed for that disease. However, complex disease causal genes are not always clear. In contrast, Mendelian disease causal genes are well-known and druggable. Here, we seek an approach to exploit the well characterized biology of Mendelian diseases for complex disease drug discovery, by exploiting evidence of pathogenic processes shared between monogenic and complex disease. One way to find shared disease etiology is clinical association: some Mendelian diseases are known to predispose patients to specific complex diseases (comorbidity). Previous studies link this comorbidity to pleiotropic effects of the Mendelian disease causal genes on the complex disease.

Methods

In previous work studying incidence of 90

SUBMITTER: Lalagkas PN 

PROVIDER: S-EPMC11385846 | biostudies-literature | 2024 Sep

REPOSITORIES: biostudies-literature