Genetic associations with diseases of aging, and knockout phenotypes for drug discovery
Approved Research ID: 60508
Approval date: July 14th 2020
We discover and develop drugs for disease of aging. We will use these data to prioritize candidate drug targets for these diseases, to identify targets encoded by genes in which genetic variants are associated with disease risk or related traits. This may provide direct evidence - in humans - that modifying the protein coding sequence, splicing, or expression of the gene can affect risk of the disease or related "endophenotypes". We will also find associations with having broken copies of the gene, as this may flag possible toxicity concerns to watch for in preclinical and clinical studies. In particular, we are interested in neurodegeneration (especially dementia & Parkinson's disease), frailty (especially sarcopenia and osteopenia/porosis), fibrosis (especially IPF, CKD, and NASH/NAFLD), senescence, immuno resilience (such as vaccine response preventing infectious disease), and caloric restriction (particularly looking at obesity).