Imagine if there was a technology that could modify your genetic code in much the same way that lines of text are cut and pasted using a word processor. It might sound like science fiction to some or Frankensteinian to others, but such a technology is on the cusp of becoming a reality, and it could actually lead to some remarkable breakthroughs, such as cures for genetic disease.
It's all thanks to an enzyme called CRISPR-Cas9 that was only just discovered a few years ago. The enzyme, which occurs naturally in some bacteria, is part of a bacteria's immune response when it gets attacked by a virus. Basically, CRISPR-Cas9 is capable of very precisely slicing and dicing viral DNA. These genetic cuts must be precise because the bacteria end up storing bits of the viral DNA so they can then recognize and better defend against similar viruses in the future.
That's great for bacteria, but it didn't take long for scientists to realize it could be great for us too. CRISPR-Cas9 is now recognized as the most effective technique for snipping and editing DNA ever found, and scientists think that by channeling this tool they can eliminate genetic disease for good.
Of course, it could also eventually lead to designer babies, but that's another matter for another time.
For now, CRISPR-Cas9 gene-editing techniques are being given the full go-ahead by investors. Bill Gates, founder of Microsoft and one of the richest men alive, is pouring money into it. He is part of a group of investors who have recently pledged to invest $120 million into a CRISPR-based start-up called Editas Medicine, a sum that will be enough to fund the company's efforts for at least three years, reports Forbes.
“They all appreciate the vast potential of this science,” said Katrine Bosley, chief executive of Editas. “The heart of the conversation we had with everybody is how you translate this very exciting but young science into treatments, into therapies.”
The first patients likely to receive the new gene-editing treatments are those suffering from Leber’s congenital amaurosis type 10, which is a genetic form of blindness. This disease makes a good test case for the technique for a couple of reasons. First, because it occurs in the eye where gene therapies are relatively easy to deliver. Second, because the genetic error that causes the disease is simple and can be easily deleted out.
Once these simpler genetic errors can be adequately corrected, the technique can be perfected for more complicated genetic maladies. If all goes well, there's no reason this technique can't eventually be applied to editing and sorting whole genomes, perhaps even rebuilding and modifying genomes from scratch. Such precise genome engineering could give scientists the power of creation itself — the ability to change the world as we know it on an ecological scale — and that's not hyperbole. The technology has even been referred to by some as a potential "Genesis Engine.”
With the massive amount of funding CRISPR projects are receiving, the future might be even closer than we all realize.