Jennifer Doudna
👤 PersonAppearances Over Time
Podcast Appearances
So really exciting, yeah.
Yeah, very good point.
So yeah, we used a version of CRISPR that was created by David Liu at the Broad Institute and published and available.
And so it was possible to create that, again, targeted to the exact mutation that caused baby KJ's disease.
And fortunately, there was also an off-the-shelf way to deliver it because we had access to lipid nanoparticles that were developed for other purposes, including vaccinations.
And the type of disease that KJ suffered from is one that is treatable by editing cells in the liver, which is where the lipid nanoparticle naturally goes.
So we were, you know, there were some, definitely some serendipity here, but it was amazing how all of these pieces were available.
We just had to pull them together to create this therapy.
Right.
Well, the IGI is a joint institute, as you probably know, Eric.
So we were founded 10 years ago as a joint institute between UC Berkeley and UCSF, and now we have a third campus partner, UC Davis, and we have the Gladstone Institute.
So we've got an extraordinary group of clinicians and researchers that are coming together for this project and this center to make it a success.
And we are building a clinical team at UCSF.
We have several extraordinary leaders, including Jennifer Puck and Chris Dvorak.
And they are both going to be involved in identifying patients that could be enrolled in this program based on their diagnosis.
And we will have a clinical advisory group that will help with that as well.
So we'll be vetting patients probably right after we announce this.
We're going to be looking to start enrolling people who might need this type of help.
I think it could be.
And here's the reason.