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CRISPR and the Augmented Human Being

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CRISPR is rapidly going to lead to transform medicine for a lot of genetic issues... but will it change our species irrevocably?

Yes it will. Today I learned there are 2000 gene therapies.

There are now 2000 gene therapies where you’ll take a little piece of engineered DNA, put it inside of a viral coat so all the viral genes are gone, and you can put in, say, a human gene or you can have nonviral delivery, but the important thing is that you’re delivering it either inside of the human or you’re taking cells out of the human and putting the DNA in and then putting them back in. But you can do very powerful things like curing inherited diseases, curing infectious diseases.

For example, you can edit out the receptor for the HIV virus and cure AIDS patients in a way that's not dependent upon vaccines and multidrug resistance, which has plagued the HIV AIDS story from the very beginning. You’re basically making a human being which is now augmented in a certain sense so that, unlike most humans, they are resistant to this major plague of mankind—HIV AIDS.

Today I also learned that the big breakthrough for CRISPR was figuring out how to do it for humans.

CRISPR was a phenomenology since 1987, but it didn’t turn into a technology until 2012-'13. It's a mechanism by which bacteria protect themselves from invading viruses by making a molecular machine that would recognize those viruses and cut their DNA, hopefully killing or at least disabling the virus. It seemed like that might be adaptable to turning it from a killing machine into an editing machine, but that transition didn’t occur, or it wasn’t public at least, until 2013. It's a molecular machine, like many enzymes, catalysts are a protein component and this one has a nucleic acid component—a relative of DNA, which is RNA. The two will then scan along your genome more or less randomly jumping around, sometimes revisiting the same site over and over that’s the wrong site. Eventually it finds the right site and when it does, it rearranges the double-stranded DNA to insert the RNA—making a triple-strand—then the protein cuts both strands. Now you’ve got a broken piece of DNA, which seems like it’s killing still not editing, but that break allows you to bring in yet another molecule, which is now your donor DNA, which now has the new sequence that you’d like to swap in. It can cause a deletion, or an insertion, or just a change of sequence with the same number of base pairs of Gs, As, Ts, and Cs; just changing the composition, the order.                                 

That’s editing. It’s just like you would edit a book or an article: you want to be precise. You don’t want to just change G to some random other thing; you want to change it to an A. And that’s the technology that we happened to demonstrate. First we moved it from bacteria into human—a huge jump—in January 2013. Then many other labs, including ours, applied it to many other organisms.                                 

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