A patient’s donated brain or lung tissue, otherwise destined for the bin, will be kept alive in the lab and dosed with chemicals to reveal what disease genes actually do inside human cells. The problem is a bottleneck. Scientists have found tens of thousands of genes linked to diseases from Alzheimer’s to COVID-19, but knowing a gene exists is not the same as knowing what it does in the right cell type under disease-like conditions. Without that information, most genetic discoveries never become treatments. The team has already proved the approach works: a gene found in severe COVID-19 led directly to a new drug. This project builds a systematic pipeline. It will use donated human tissue—brain, lung, skin—that would otherwise be discarded, expose it to chemical cocktails that mimic real disease environments, and track how genes and cells respond. The data will be shared openly and rapidly with the global research community. This is fundamental science with a clear translational track record. If it succeeds, it will turn a flood of genetic leads into a shortlist of druggable targets, accelerating drug development for common and rare diseases alike.
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Modern medicine enables doctors to treat diseases better than ever before, but there is still much more to learn. Human genetics can help. Tens of thousands of genes have been found underlying a number of devastating diseases, from Alzheimer's to COVID-19. These genes sometimes gives us clues that can help everyone with that disease, not just people who have particular genes. We have already shown that this works: we found a gene in severe COVID-19 that led directly to a new and effective treatment. To make the jump from disease gene to treatment, we need better information about what disease genes actually do in the right cell types from the human body, and in conditions that mimic real disease. Our plan is to set up a system to study how genes work in hard-to-get cell types and conditions. We will use real human samples, donated by our patients, that would otherwise be discarded. We are already able to do this with samples of brain, lung, and skin. To mimic the complicated environment inside a patient even more closely, we will then add a range of different chemicals to the samples and see what happens to the genes, and the cells. We will share our data quickly with scientists across the world to move medical research and drug development forward. We will build a community of doctors and scientists to help guide us towards the most important and useful gene functions. Together, our system will give much more information about genes and how they work to make disease better or worse, giving us a chance to find better and more accurate treatments to help more patients.
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