CRISPR/cas9: the revolutionary gene-editing tool

By March 7, 2017Blog Posts, Pharma Beat

In the 1980s it seemed absurd to think computers would change the world. Could we be at a similar point today with gene editing?

The latest discovery in gene editing is a component of the prokaryotic immune system which can be programmed to modify any sequence of DNA from any organism. Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR associated system 9 (cas9) form a gene editing tool (CRISPR/cas9) that is accurate and easy to use. Potential applications of this technology range from curing disease to creating designer babies.

There are many ethical and technical challenges to address before gene editing can be used as therapeutics or for genetic modification, however CRISPR/cas9 has already proven itself useful in the laboratory. Genetic engineering is now faster and less expensive, making it easier to study cellular systems and the molecular basis of disease.

Research into developing CRISPR/cas9 as a therapeutic tool is currently underway. In the US, 18 patients will have their T-cells edited to better detect and fight various types of cancer. China has approved a small clinical trial to investigate gene editing in patients with metastatic non-small cell lung cancer and has also conducted gene editing research in human embryos. Editas Medicine Inc., a genome editing company in the US, is currently developing CRISPR/cas9 to treat genetic diseases such as Duchenne muscular dystrophy and cystic fibrosis. We eagerly await results from these studies, which will provide valuable insight into how CRISPR/cas9 will impact the future of healthcare.