Genome Editing Trials

For the first time, the exciting gene-editing tool CRISPR-Cas9 has been used in a human. This technology, which I wrote about several weeks ago (refer to post from Nov. 5 for more detail), allows scientists to alter DNA bases to introduce a change to any gene. The concept of altering human DNA is controversial, but the potential health applications are practically limitless. For example, if a disease is caused by a specific, known mutation, scientists could potentially alter that mutation, thus treating the disease. This technology is a hot topic among biologists, and is also currently one of the most-studied tools.

Cancer is one of the main diseases that scientists hope can be treated using CRISPR-Cas9. Because of this, the first use of CRISPR-Cas9 has been in a human patient with aggressive lung cancer. Scientists of the West China Hospital in Chengdu have altered a gene that normally prevents a cell from launching an immune response, so that the immune system is more likely to target and destroy cancerous cells. The researchers hope that after injection into the patient's blood stream, the modified cells will then start attacking the cancerous cells. 

While this Chinese trial, the first of its kind, began in October of 2016, the United States also plans to start a human trial of CRISPR-Cas9 early this year. The U.S. trial is also testing the safety and efficacy of using CRISPR technology to combat several forms of cancer. Since both China and the U.S. are launching CRISPR-Cas9 trials, some healthy competition is predicted to fuel the potentially life-saving research. Carl June, a scientific adviser to the U.S. trial, says "I think this is going to trigger 'Sputnik 2.0,' a biomedical duel on progress between China and the United States, which is important since competition usually improves the end product."

To watch a video and gain more information about this Chinese trial, follow this link:

http://www.usnews.com/news/national-news/articles/2016-11-17/gene-editing-tool-crispr-cas9-used-in-a-human-for-the-first-time