Genome Editing

So many diseases come from defective genes — but what if we could just replace those defective genes? That’s the idea behind genome editing, which has been gaining a lot of steam in recent years. With this technological advance comes a major opportunity to wipe out tons of genetic diseases.

Many biotech companies have been successfully gathering funds for research on this possible treatment, including Bluebird Bio, which had a $116 million IPO recently, and Juno Therapeutics, which has raised $120 million in venture capital.

The ability to edit or replace specific genes relies on modified viruses like the adeno-associated virus (AAV), which can get into a person’s body and replace the faulty genes with good ones. Another method is to remove some cells, treat them with a modified virus, and then reinsert the cells back into a patient’s body. All of these methods could prove to be quite expensive, but as more companies invest in it, the price should go down.

Genome editing — or gene therapy as it’s also called — differs a bit from genetic modification, although more so in purpose than in application. Both alter the genes in an organism’s genome, but genome editing seeks to fix defective genes while genetic modification seeks to change well-functioning genes to improve an organism. This is great for increasing the nutrition or improving the taste of food, but when it comes to humans, the ethics of “improving” them is scary.

As long as we stick to curing diseases rather than “improving” ourselves genetically, this emerging technology is wonderfully exciting.