Name: Here’s What You Need to Know About Gene Editing
Uploaded: 2016-11-02
Duration: 4 min 35 s
Description: Dr. Rachel Haurwitz explains.

[ MUSIC ] [ UNKNOWN ] gene editing is taking the life sciences by storm right now. Gene editing is basically the biological equivalent of a word processor. It allows researchers to take genetic code And precisely insertly or change of nuclear strands in a way that we might insert or change a few words in a text file. The first paper showing how this casper caspian technology worked was published in 2012. And since then thousands of labs have adopted this technology. It's been credited for democratizing gene editing. Many of these labs are incredibly excited, because it gives them the ability to better recapitulate disease causing mutations, in both cellular and animal models of disease. Giving researchers better insight into development, disease biology, and hopefully ultimately giving them the tools To develop better drugs for a number of diseases. But a huge amount of this excitement comes from the potential to directly use this technology to treat and potentially even cure genetic diseases. Take for example sickle cell anemia. It's a disorder that's caused by a single mutation in the hemoglobin chain. And results in patients having red blood cells that look like little sickles. And they don't do a very good job of carrying oxygen. In theory, a doctor could remove a patient's own blood stem cells, take them to the laboratory, and using Crisper Cast 9 correct that single mutation. And then transplant those corrected stem cells back into the patients providing them normally functioning red blood cells. There's a tremendous amount of work yet to be done to fully translate this technology into genetic medicines. And there are a number of companies and labs around the world actively doing this. The first handful of trials testing this technology in human in both genetic disorders and cancer treatments will kick off this year, next year, and the year after that. But it's not just humans who will benefit from this technology. Our company recently had the opportunity to join a collaboration. Where researchers are looking to breed pigs that are resistant to the porcine reproductive respiratory syndrome virus. It's a mouthful I know, PRRS. And it's the worst virus farmers face in their pig population today, for which there is no cure. Our collaborators have already shown, That by knocking out just one single protein in the pig genome it can generate pigs that are completely resistant to this virus and otherwise healthy and happy. Again, there's a lot of work to be done to turn this into a full commercial product but it's a really exciting potential case for ways to treat animal health and well being issues. And at the same time increase global food security. And finally, there's tremendous potential to use this technology in the generation of crops to continue feeding our growing global population. For example, a recent academic project showed the ability to generate a mushroom through CRISPR- Cas9 editing. That doesn't brown as quickly. And in theory, products like this will have a longer shelf life and less food will go straight into the waste stream. A number of companies and labs around the world are using gene editing to better understand plant genetics. And ultimately develop crops that can be drought tolerant, diseases resistant, and maybe even allergen free. So, how does all this work? Cas9 is a protein that's basically a pair of molecular scissors, and with the help of a guide RNA, it can actually target and cut DNA at the exact site where the researcher wants to insert or delete DNA nucleotides. Now it turns out, cells don't really like to have their DNA broken. So when that happens, they quickly move to fix it. And depending on how it's fixed, there a variety of different edits that are possible. So if this sound intriguing to you, and you're curious about the various applications and the ethical implications of technologies like this Please join us for the synthetic biology discussion at breakfast tomorrow. Thank you. [ APPLAUSE ] [ MUSIC ]