Gene Editing

DNA is the instruction manual for life on Earth. It encodes the fundamental properties of an organism and is passed down from cell to cell, from generation to generation. Most of the time, this happens without any issue. In some cases, however, mistakes occur during the process, leading to changes in the DNA sequence. These might be without effect or harmless, but they can also cause one of the thousands of rare, monogenetic, and sometimes life-threatening diseases.

What is gene editing?

With technologies like CRISPR, that build on Nobel Prize winning science, gene editing allows for the targeted manipulation of genetic material, making it possible to correct such changes in the sequence when they cause disease. While gene editing is one of the youngest of the CGT approaches, the technology is advancing rapidly, meaning that novel therapies might be just around the corner.

Science and Technology

In healthcare, gene editing technologies enable the precise manipulation of genetic material to achieve a therapeutic effect. This includes a wide range of applications such as the correction of mutations that cause disease, the addition of therapeutic genes to specific sites in the DNA, and the removal of deleterious genetic sequences. Because of this diversity, one can think of the technology as a Swiss army knife – an invaluable tool with a diversity of functionalities. For healthcare applications, gene editing has a dual role: it is used as an enabling technology for cell therapy and also has huge potential as a stand-alone therapeutic technology.

As an enabling technology for cell therapy, gene editing can help to alter and therefore improve therapeutic cells, which will allow the development of next generation cell therapies with increased safety and/or efficacy, for example, increased potency and persistence or reduced immunogenicity. This could enable cell therapy application to a larger patient pool or a broader indication range. Therapeutic gene editing, on the other hand, is a  specific type of gene therapy that is just starting to emerge. Research is ongoing to create such gene editing therapies that can combat cancer, blood diseases, infectious diseases, and rare inherited genetic diseases. One example of a gene editing technology is CRISPR, which is currently being explored in clinical trials as a therapeutic modality for diabetes, sickle cell, and many other diseases. 

The ability to manipulate DNA with unrivaled precision makes gene editing one of the most versatile approaches to treating diseases – and an approach that has the potential to constitute a paradigm shift in healthcare.