Gene editing technologies leading to medical and biotech breakthroughs
Gene editing, also known as genome editing, is a technology that allows scientists to make specific, targeted changes to an organism’s DNA. This involves adding, removing or altering genetic material at specific locations within the genome. In the field of medicine, genetic therapies have the potential to be used to prevent, treat or even cure certain inherited disorders. Researchers are also looking into how they could be used to treat cancers and infections. Beyond that, the more we learn about DNA, the more we get a glimpse of how living things function. Studies into DNA have shown us how cells grow, divide and repair, and how critical processes in the body, such as metabolism and immunity, are controlled at the genetic level. Looking into the distant past, DNA studies can also reveal a great deal about migratory patterns and ancestry. And looking to the future, gene editing potentially opens the door to the engineering of synthetic organisms designed to perform specific tasks. Robots consisting of living cells could be made to be self-powering and self-healing, performing functions that would otherwise be hard to engineer from inert material.
Addressing key medical and societal issues
This Pack presents a diverse portfolio of projects supported by the European Research Council(opens in new window) (ERC), which demonstrate how gene editing is helping us to address key medical and societal issues. The ERC is dedicated(opens in new window) to funding frontier research that pioneers the development and use of groundbreaking gene editing technologies. One of these, CRISPR/Cas, has opened new possibilities in both basic science and applied research, enabling researchers to tackle previously unanswerable questions. Over 30 ERC-funded CRISPR/Cas projects have resulted in patents, with some leading to the creation of spin-off companies. The ERC-funded projects highlighted in this Pack bring together researchers from a range of disciplines including molecular biology, genetics, chemistry and bioinformatics, to name but a few. Together they demonstrate how a focus on basic research can deepen our understanding of biological mechanisms and human diseases, while opening up practical applications in agriculture, biotechnology and medicine. The knowledge generated through this groundbreaking work will also help policymakers and stakeholders to better understand the benefits and potential risks associated with gene editing, and to take supportive action accordingly. EU-funded researchers have made significant advances in unravelling the secrets contained within DNA, and how the potential of gene editing can be harnessed to bring health benefits. At a fundamental level for example, ERC-funded projects such as MIGHTY_RNA have pioneered new methods for observing molecular interactions. LoopingDNA has shown how chromosomes are fundamentally structured in DNA loops and provided a deeper understanding of the mechanisms behind this process. Similarly, CHROCODYLE has demonstrated how this looping process is critical for ensuring chromosomes are properly distributed during cell division. MaCChines meanwhile has pioneered the design of new proteins, which could potentially open the door to new breakthroughs in medicine, technology and science. RESOLUTION managed to sequence the genome of multiple individuals from a remote Neanderthal community in Siberia, providing a glimpse into their social organisation and giving us the most accurate timeline to date for when our ancient ancestors met their Neanderthal cousins. When it comes to addressing cancer, ConflictResolution has developed a toolbox to study cellular conflicts in a breast cancer genome. EpiFold has built on evidence that tumours exploit the functions of non-cancer cells in their microenvironment to invade and metastasise. And finally, GWAS2FUNC has helped to clarify the genetic variants present in a range of diseases, which could improve clinical practice and future drug development.