You don't know what your future will be.
You don't know what will happen. It could be fine, I could be healthy,
but I could also end up in a wheelchair. I know what I want to be when I grow up
and all that. But you don't know
what you'll still be able to do. So really it's just enjoying every day
to the full. Every day is another day. Oh dear, I don't have a parachute.
So there I go, falling to my death. If you have a parachute, you can use it.
But apparently, I didn't have one. The earlier it starts, the worse it will get. And the shorter the chromosome,
the more serious the disease. So Ivan is not in luck. He's very active, which is good. It's been
proven that exercise is good for you. But he'll need medication within ten years
in order to be all right. So it's a race against time. What will come
first? His deterioration or new drugs? Holland played an important part, or at
least Wageningen University or my group. Because we were one of the first
to discover some very basic principles. I'm pretty proud of that. It's fantastic to see this being used
by all kinds of groups all over the world. It's a real revolution in biotechnology. From now on, everything can be done
much faster and more efficiently. That makes a huge difference. Not just in biotechnology,
but in medicine as a whole. The CRISPR system
has broad applications. You can use it for genome editing... ...to change the genome
of a bacterium or yeast cell. That's often biotechnology. So you use microorganisms
to make certain interesting products. It's a real biotechnology revolution. So from now on, everything can be done
much faster and more efficiently. That makes a huge difference. Not just in biotechnology,
but also in medicine. Do you want something to drink?
Go grab a glass. It sounds a lot like science fiction. But if it works, he could be cured
of this disease in one fell swoop. So it's promising... ...and very interesting.
This is the age of science, of course. You know a bit about how it works,
what he does? Not really, no. They're cutting a chromosome, right?
You know how your disease started. Because of a shortened chromosome.
We can ask him about it. Don't we need to wear lab coats?
- Not here, no. This is the molecular lab,
where we work with molecules. The most important molecule
we work with here is DNA. Here we can prepare genes... ...so bits of DNA which code
for a certain protein... ...for incorporation in certain cells
which then lets us study that gene. One of the genes we're studying
is the DUX4 gene... ...which is causing you such problems.
- You know that, right? That gene is switched on in your muscles,
but it should be switched off. Because it's on, your muscles
are getting weaker and weaker. But we'll do our best.
- Anything you want to see? Shall we have a look at some cells? The problem is that a protein
is being produced in his muscle cells... ...which shouldn't be produced at all,
because it's very toxic. So very slowly, the muscle cells
in his face, neck and shoulders... ...and maybe other parts as well,
are dying. That means he's growing weaker,
his expression is rigid. It's hard for him to show emotion, or smile.
That's what is going wrong. You see those bumps, those round things. Those are cells which have died,
or which are dividing. Simply put, with CRISPR-Cas we'll try
to make a cut in the DNA somewhere... ...in order to shut off the gene, the toxic
protein, causing all these problems. With CRISPR-Cas we can do things
that were impossible a few years ago. For example, genetically adapting cells
in the lab... ...so we can mimic certain DNA defects
we see in patient cells in the lab. A few years ago, such a project
would take a PhD student four years. Now, you can do it in two weeks. So it's led to a huge acceleration
of research... ...meaning we can find out much more
in a much shorter time. That in and of itself is revolutionary. What will this acceleration mean? With every new technology being
developed, in this case it's CRISPR-Cas... ...but there's also genome sequencing,
determining a DNA sequence... ...or technologies allowing you to see
things you could never see before... ...you get an acceleration of knowledge
and also of applications in medicine. But it will still be a long wait before
patients will finally reap the benefits. Success in our laboratory means... ...we can now do something
we couldn't do yesterday. But between that discovery,
which has got us cheering... ...and the development of such
a technology into a workable therapy... ...another 15, 20 years could pass. Boston is a fantastic place, a real hotspot. You have MIT, Harvard, and all kinds
of institutions, like the Broad Institute. There's a lot of money, great people...
It's the cream of the crop. I'm in Boston to visit some people. I was
invited by Feng Zhang at MIT Broad... ...and Keith Young
of Harvard Medical School... ...in order to give a talk,
so that's what I'll be doing. I'll also be visiting Editas,
where I've been before. They make frequent use of Cas9,
but also Cas12 or Cpf1... ...for their applications. So we'll be discussing
the progress of my research... ...the progress of their own research... ...and I'm curious to see
whether we can work together. A large number of companies are using
CRISPR technology by now... ...in order to tackle human diseases. I don't know what all those companies
are working on. They won't always tell you. But if you
look at Editas' website, for example... ...you'll see they're focused
on eyes, blood, cancer and liver. Of course, they'll want to make money
from it at some point, like any company. They regularly raise funds, so they now
have a team of over 100 people. You can only do that
with external funding. Do you have any idea how much?
- I have no idea. But you sometimes read online that
they've had another successful round... ...and that's rounds of 100 million dollars.
So a significant amount. There's a good chance that
those companies will be very important. If you look at what Apple and Google
have achieved in IT... ...companies like Editas and Caribou
and others are their equivalent. They're working hard now... ...to get some very important work done
in the field of biology... ...and to put that technology
on the market. Is there a risk of them becoming too
powerful, like Apple and Google? I couldn't really say. Of course, it's a good idea to monitor that
anyway. There should be regulations
to set the limits. So... When? That's an important question.
- When will he be fixed? It's hard to predict how long it will take. There have been discoveries where
people thought: This will take years. And then, all of a sudden, it happens.
Sometimes the result seems so close... ...but it stays just out of reach.
So it's very hard to say. Let's hope, then. Work hard.
- We'll do our best. Anything you want to know?
- I know everything. That's good, you know everything.
