Eric Topol
๐ค SpeakerAppearances Over Time
Podcast Appearances
And that is so one of the biggest reasons why this is a big step forward and why we're so fortunate that your center is moving forward.
Maybe before we wrap up, you might want to comment, Jennifer, on
how you were able to bring in, to build this platform, the manufacturing arm of it, because that seems to be yet another dimension that's helpful.
Oh, that's phenomenal because some of these disorders you don't have that much time to work with before they could be brain or organ or vital tissue damage.
So that's great to hear that.
What you built here is the significance of it can't be under-emphasized, I'll say, because we have this May report of baby KJ, which could have been a one-off.
And it could have been years before we saw another cure of an ultra-rare disorder.
And what you're doing here is insurance against that.
You're going to have many more cracks at this.
And I think this is the excitement about having a new dedicated center.
So just in closing, some remarks from you, Priscilla?
And over to you, Jennifer.
For sure.
Now, if I could just sum up, this is now a decade past the origination of your work of CRISPR and how already at the first decade culminated in sickle cell disease treatment and beta-thal.
Now we're into the second decade of CRISPR and look what we've seen.
Something I didn't think would be, it was unimaginable until it actually happened and was reported just a little over a month ago.
Now, going back to Priscilla's point, we're talking about thousands of different rare Mendelian genomic disorders, thousands of them.
And if you add them all up of rare diseases, we're talking about hundreds of millions of people affected around the world.
So this is a foray into something much bigger, no less the fact that some of these rare mutations are shared by common diseases and approaches.
So this is really big stuff.