Rare Diseases

Surat Parvatam, Francesca Pistollato, Lindsay J. Marshall, et al. Human-based complex in vitro models: their promise and potential for rare disease therapeutics. Frontiers in Cell and Developmental Biology. 2025;13. doi:10.3389/fcell.2025.1526306

Katerina Trajanoska, Claude Bhérer, Daniel Taliun, Sirui Zhou, J. Brent Richards, Vincent Mooser. From target discovery to clinical drug development with human genetics. Nature. 2023;620(7975):737-745. doi:10.1038/s41586-023-06388-8