A logical network-based drug-screening platform for Alzheimer's disease representing pathological features of human brain organoids.

ABSTRACT: Developing effective drugs for Alzheimer's disease (AD), the most common cause of dementia, has been difficult because of complicated pathogenesis. Here, we report an efficient, network-based drug-screening platform developed by integrating mathematical modeling and the pathological features of AD with human iPSC-derived cerebral organoids (iCOs), including CRISPR-Cas9-edited isogenic lines. We use 1300 organoids from 11 participants to build a high-content screening (HCS) system and test blood-brain barrier-permeable FDA-approved drugs. Our study provides a strategy for precision medicine through the convergence of mathematical modeling and a miniature pathological brain model using iCOs.

SUBMITTER: Park JC 

PROVIDER: S-EPMC7804132 | biostudies-literature | 2021 Jan

REPOSITORIES: biostudies-literature