Breakthrough gene editing treatment helps child born with rare disorder

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Dr. Peter Marks:

Yes, I think that’s a really important discussion to be had.

And I think one of the reasons why this technology is so exciting, at least to me, is that it has the potential to bring down the cost of gene therapy, which people might know, many of the gene therapies now are in the millions of dollars, a whole order of magnitude.

So this is in the — hopefully, first — the next step will be into the hundred thousand of dollars and then hopefully into the tens of thousands of dollars. Could be a huge game changer, because we could see these types of gene therapies making their way into low- and middle-income countries, where they could essentially leapfrog care and help people, for instance, with sickle cell disease, where, right now, there’s just nothing for them.



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