CRISPR and You

Delivered on 6/26 in Dallas

 
 

By a show of hands, who here could tell me what CRISPR CAS 9 is? Not a lot. That's good. By the end of my 7 minutes, everyone will know what CRISPR is and how it works. Let’s build up to that and start with DNA.

DNA is a language (like English or Spanish) used to write the instruction manual of how to make you. That instruction manual is called the genome. It contains the step-by-step directions needed to make your beautiful eyes, your blood vessels, your spleen, everything! In fact, everyone here has a slightly different genome, and that's what makes us different. And every living being has their own genome too. Isn't this fascinating?

The United States government spent 13 years and 2.7 Billion dollars to completely read and write down the first human genome. That was completed in 2003. This technology has gotten exponentially better and cheaper since then. THIS is MY personal genome, every instruction on how to make a Dalmo, and I got it for 99 bucks. Let’s read what’s inside, shall we?

With several genomes available, we can compare them and understand what pieces do what. As you’re reading what’s written in my DNA, this section, this gene, is responsible for my deep brown eyes. This one for healthy and plump red blood cells. This one got damaged somewhere, so it causes my asthma.

So as you can see, we've gotten really good at reading the genome. There are pieces of it that we even understand. Knowing where my asthma comes from is pretty neat, but it doesn’t mean I can do anything about it. I can’t make changes to my instruction manual.

Let me tell you the story of a couple of brilliant women, Dr. Doudna and Dr. Charpentier. They were researching the Streptococcus genome in 2012. Streptococcus is the bacteria that causes those nasty strep throat infections. As they were reading it, they came across something interesting. First they found some short palindromes. Does everyone know what a palindrome is? It’s a word that is the same read forward or backward, like RACECAR. Then they realized the palindromes kept repeating, over and over and over again, but they were interspaced with some genes with no known function. Palindromes, a gene, more palindromes, another gene, etc.

This seemed important, so the scientists did two things: 1st they named it: Clustered, Regularly Insterspaced, Short Palindromic Repeats: CRISPR. Then, they watched to see what it did.

It turns out, one of the genes was the instruction for a big protein equipped with some sharp cutting parts. They called CRISPR-Associated System #9, or CAS 9 for short. The other genes were instructions to make these little templates that fit right inside of the CAS 9 machine.

The researchers watched and they saw a virus infecting the bacteria with its own short little genome. As soon as the virus entered the bacteria, CAS9 jumped on the virus genome, attached to it with the template, and chopped it to pieces. This whole CRISPR system was the bacteria’s defense mechanism: it stored previous infections as templates so that CAS9 could target new infections.

Here’s the genius of Dr. Doudna and Dr. Charpentier: they saw the potential of CRISPR as a gene editing tool. With this tool, for the first time, we can quickly and accurately target a specific part of the genome to remove it, fix it, or change it.

Now that you know how CRISPR works, let me share with you a few questions you might ask. First, the ethical questions: Should humans have this much control over nature? Or are we playing God with our bodies? What if people start editing genes so they have different color eyes? Or stronger muscles? Are we okay with that? And when this technology comes to market, who’s going to have access to it? Only the rich? Will we be able to design our babies to be healthy or unhealthy, short or tall? And should we do that without their consent? And now that we can edit the genome, who OWNS your genome? Is it your property? Or is that in the public domain? These questions remain unanswered.

But now let’s think of the possibilities. What if I can go back to my genome, snip out the damaged piece responsible for my asthma, and replace it with healthy instructions? What if we can reverse the causes of sickle cell anemia, radiation poison, most types of cancer, ALS, and even HIV infections? Or save species from going extinct by engineering their genome to make them more resistant? What if we could edit the mosquito genome to make sure they can’t transmit malaria anymore? Some of this is already happening right now, and many other possibilities are in the horizon.

As I come to a close, think of the following words: Vaccines. Chemotherapy. In Vitro fertilization. At one point, these were words people were unfamiliar with. Nowadays, we know them because they're so widely used. I think the same will happen to CRISPR. And in a few years, when those questions and possibilities become a reality for you, I hope you'll be equipped with this knowledge to make your own informed decisions in this brave new world. Thank you.