Neville Sanjana, faculty member at NY Genome and New York University, joins Cheddar Reveals to break down the latest innovations in CRISPR genetic engineering and its applications from 'designer babies' to curing genetic conditions and diseases.

Cheddar Reveals

Editing the Human Genome

Welcome back to Cheddar reveals, brought to you by curiosity stream every day. There are innovations and advancements that blur the lines of what we think is science fiction and what is reality. One of the biggest growth areas is the realm of genetic engineering which could have applications from everything to curing cancer to creating a real life drastic park. Joining us now is Dr neville sanjana core, faculty member at new york genome center and assistant professor of biology, neuroscience and physiology at N. Y. U. DR. Let's just kick things off by diving into crisp or what is it at its core. And what are the applications? Yeah thank you for having me on CRISPR is a gene editing technology. You can think of it like cut copy and paste but for D. N. A. And D. N. A of course is the code that runs you as an organism. And on a molecular level, CRISPR itself functions kind of like a pair of scissors it cuts in a very specific place. Which is challenging because the human genome is huge. And there are many applications for CRISPR right now that are being developed biomedical research to understand the genetic basis of disease new therapeutics to treat those diseases sensors to detect viruses like SARS cov two that causes Covid and editing important food crops to give them resistance to pests or drought and ensure food security. When it when we look at CRISPR and the potential role that it plays in the future of condition and disease treatment. What is that Exactly. Yeah that's that's a nice question. So we don't really need to look to the future, CRISPR is already having an impact on human disease. So take sickle cell anemia which is an inherited blood disorder of red blood cells. Basically they produce a mutated form of a protein called beta globe, very painful disease um found throughout the United States. Asia africa. Found in many places in work we did a few years ago with stu orkin and dan Bauer at Harvard. We found a short piece of D. N. A. That when it's edited in folks with sickle cell it lets them substitute another copy of the hemoglobin gene for the defective adult beta global. And this therapy is now in human clinical trials and early results look very promising, wow. The idea that we can go from that to to even other diseases and really help modify and maybe even eradicate some of some of the harmful diseases that people die from every day. I wonder in terms of genetic engineering as a predictive tool we can talk about that. Can we see what diseases a person may be susceptible to and then maybe put in preventative measures in place you know for them to prevent them from happening altogether. Yeah, this is a really interesting idea. Um but the truth as a human geneticist is that it's really hard to predict exactly what diseases a person will get just from their genome. And there's a few examples of um kind of very rare serious diseases that are caused by one region of the genome. I mentioned sickle cell anemia just a second ago. But when we talk about common diseases, diseases like heart disease or diabetes, the genetic contributions are spread out over the entire genome. Um I personally am very hopeful that the in the future the human genome will be easily interpret herbal and by reading it we can understand a lot about disease risk just from the D. N. A. But we're not really there yet. We are far away from there. And this is one reason why human genome research is so important Right now when I hear about this these developments I obviously think about a lot of the sci fi movies that we've seen a drastic park you can name. I'm sure a ton of them that points in maybe some of the dangers or ethical challenges around this. But let's talk about the real life kind of dangers and ethical challenges associated with genetic engineering. Yeah like any new tool in biology or otherwise gina editing must be used responsibly. And right now the field is focused on using it to help patients with serious genetic diseases. So most scientists and clinicians are really focused on what we call somatic gene editing which means editing specific cells tissues or organs in adults. Um And these r right now very new approaches to curing genetic diseases are not things that are widely used are really used at all. But hopefully in the future will become routine and improve millions of lives. Now there's another kind of gene editing which is called Germline genetic, this is editing sperm and egg. And it's quite a serious thing if you think about it because not only are you making the edits in an individual, but potentially in future generations. So in addition to kind of the numerous ethical problems of doing this, it really is just not needed. Is the simple answer. Right now, we have many kinds of tests to diagnose rare genetic disorders in parents who might be carriers of these diseases or using non invasive blood tests. Um in the mob Really quickly here, I've only got like 20 seconds but I'm curious how how far down the line do we actually see something like crisp or more widely available? Yeah. So CRISPR technology really is in its infancy right now. Its use in human cells is about 10 years old. Imagine computers, you know, in the 19 forties or fifties and that's what you have right now. Imagine entity but we're making very rapid progress. There's a few major challenges that I see one of them is precise editing with CRISPR. It's pretty easy to make an edit of some type but it can be a little bit hard to make a precise edit to put the exact letters you want in the exact place you want. And the second challenge is delivery. Even if we have amazing gene editing tools, they're useless that we can efficiently deliver them to the right cells or tissues. So even though it's early days, I think the future is very bright for self therapy and gene therapy were so certainly hopeful of the future is right. Dr really appreciate your time, dr, neville sanjana core faculty member and new york genome center and assistant professor of biology, neuroscience and physiology at N. Y. U.