
Gene Editing Tool Hailed as a Breakthrough, and It Really Is One - evo_9
http://www.npr.org/sections/health-shots/2015/12/28/460705645/gene-editing-tool-hailed-as-a-breakthrough-and-it-really-is-one
======
reasonattlm
Something to consider: it is now technically possible and within the grasp of
hundreds of clinics worldwide to run gene therapy. The only reason they aren't
yet doing this is that it takes some increment of time to get things lined up;
funding, customers, propagation of knowledge, etc. But all the pieces are in
place.

Myostatin or follistatin gene therapy is about as proven as these things get.
It exists in heavily muscled natural mutants, including humans (look up brute
whippets). Antibody myostatin blockade has just completed phase 2 clinical
trials, adding muscle mass to 75 year olds. Chinese labs are turning out
myostatin-free dogs. The BioViva CEO organized her own follistatin gene
therapy earlier this year in an overseas lab. Five years from now it won't
take starting a company level of networking to fly somewhere and do this.

But myostatin for muscle mass is just the best risk/benefit/knowledge picture
out of hundreds of alterations that could in theory now be run up in humans,
many of which are high risk in the sense that they've only been tried a few
times in mice and not followed for a long time. For example adding additional
lysosomal receptors to mice restores liver function in old mice to be the same
as in young mice. Increased GDF-11 sends stem cells back to work and improves
health in old mice.

And so on and so forth. The eager early adopter with a hundred thousand
dollars to spend ten years from now could spend the time between now and then
mining the research literature and organizing collaborators, making
connections with labs overseas and setting up for multiple gene therapies down
the line, many of which are associated with interventions shown to delay or
turn back specific measures of aging in mice.

This isn't SENS, it isn't repair of underlying damage, but it is a step ahead
of what is currently possible.

~~~
jforman
The greatest barrier here is the FDA. They don't let you perform medical
interventions (even breakthroughs like this) without going through a rigorous
process. Companies like Editas ($43M Series A) are going through those motions
right now.

~~~
mkempe
The inevitable consequence will be either the abolition of the FDA; or a chasm
between advanced medical therapy readily available in some places outside of
the US and sclerotic, bureaucratic "healthcare" in the US.

Long-term, where I live will depend on the above outcome.

~~~
jforman
CRISPR is far from being the first technology to be held up in the United
States while being available elsewhere. There is very little chance the FDA
will materially budge on their standards, and even less chance the FDA will be
abolished. Most people have a high degree of trust in the FDA (they have a 65%
favorability rating according to Pew).

Note that the FDA does allow some compassionate use of experimental
medications and technologies in extreme cases. I'm not familiar with Editas's
regulatory strategy but it wouldn't be crazy for them to pursue that avenue
for, say, a child cancer patient as an early step. But even they will be
cautious, as any misstep (i.e., harm to a patient) will have major negative
consequences for the technology as a whole.

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fasteo
Although not usually mentioned, it is important to note that CRISPR is a
natural process that bacteria use as part of their immune system. For what is
worth, the mechanistic process and a specific functional result - defend
against viruses - have stood the proof of time.

"bacteria use Cas enzymes to grab fragments of viral DNA. They then insert the
virus fragments into their own CRISPR sequences. Later, when another virus
comes along, the bacteria can use the CRISPR sequence as a cheat sheet to
recognize the invader." [1]

[1] [https://www.quantamagazine.org/20150206-crispr-dna-editor-
ba...](https://www.quantamagazine.org/20150206-crispr-dna-editor-bacteria/)

------
smoyer
It's funny that this should appear on HN ... Sunday I just completed a bit of
software that looks for candidate CRISPR sites in a particular mouse genome
for my daughter. In our case, the real trick is to find repeats in the section
of the chromosome she wants to manipulate that don't also exist in the rest of
the chromosome - or any of the other chromosomes.

Like any "big data" project, the trick is representing your data in a way that
makes searching it trivial as well as parallelizable.

~~~
junto
That sounds like a very interesting and I'm assuming, a sad story. Do you mind
if I ask if you are sharing it publicly?

~~~
smoyer
It took me a few rereads of my original post to realize what might be sad
about it ... then I got it.

It's actually a happy story (for me). My daughter is a PhD candidate at the
Johns Hopkins school of medicine and is working towards becoming a researcher
(molecular genetics). I'm not sure I should comment on which lab she's working
in or what they're trying to accomplish.

It might not be fun for the mouse but I'm thankful for projects like this
because we get to work on something together. It's almost like going back to
when she was a girl doing a science project in elementary school.

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miiiiiike
Radiolab had a great episode about CRISPR earlier this year:
[http://www.radiolab.org/story/antibodies-
part-1-crispr/](http://www.radiolab.org/story/antibodies-part-1-crispr/)

------
MathsOX
A quote that stuck with me from a molecular biology class taken a year ago,
"It's not who wins a Nobel Prize off of CRISPR work, it's how many there will
end up being".

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leeoniya
From what I understand (from a PhD in biotech), the price for utilizing
CRISPR/Cas9 is extraordinary. So much so that only multi-million dollar labs
can afford to use it in any meaningful quantity. The price "per reaction" is
in the thousands.

~~~
zzalpha
Any insight into the source of those costs?

~~~
leeoniya
licensing the tech, plus no competition = charge what the market will bear

[http://labiotech.eu/crispr-patent-war-end-discovery-new-
edit...](http://labiotech.eu/crispr-patent-war-end-discovery-new-editing-
protein-cas9/)

~~~
zzalpha
So patents. That was going to be my guess.

So in 20 years it'll be pretty cheap. Meanwhile, China and other parts of the
world that happily ignore patents will use the technology without restriction.

~~~
mentat
There's a common sci-fi theme about China being way ahead in the future. I've
never heard a plausible explanation in book but ignoring patents is a really
interesting angle.

~~~
CamperBob2
That's how it always works. Early American industrialists weren't exactly
diligent at paying royalties to British and continental European patent
holders... and now it's China's turn to prosper by doing the same thing to us.
More power to them.

Patents are a racket that governments run against their own people,
exclusively for the benefit of a very few inventors and a whole lot of
lawyers.

------
entee
I've often wondered how CRISPR is actually useful, because at its core it's
just a nuclease. It allows to you precisely cut DNA where you want to cut it.
But for that to be useful you still have to put the DNA back together
afterwards. I found this article useful for understanding the different ways
that happens in practice:

[https://www.addgene.org/CRISPR/guide/](https://www.addgene.org/CRISPR/guide/)

Turns out you get some small mutations in the most common mechanism (non-
homologous end joining or NHEJ). This could be a problem for use in therapies,
but definitely not a huge issue for most research-based applications where you
can control which mutants you allow to proceed to the next experiment.

~~~
88e282102ae2e5b
This is one reason why Cpf1 could be interesting, since it leaves sticky ends
when it cuts:
[http://www.cell.com/cell/abstract/S0092-8674%2815%2901200-3](http://www.cell.com/cell/abstract/S0092-8674%2815%2901200-3)

------
thecosas
I am excited and worried about what CRISPR will bring about.

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dekhn
The application of CRISPR/Cas to gene editing, while a breakthrough, is more
about technology than science. It just makes things easier- similar to the way
compilers make it easier to program computers than assembly language.

I also find it interesting that we consider this tool a breakthrough when
nearly all our tools for gene editing are just copped off bacteria that have
been using them for billions of years.

~~~
zzalpha
Yeah! Like, antibiotics, man. It was basically mold that invented that!
Totally not a medical breakthrough at all... no idea why so many people were
impressed by it.

~~~
bostonpete
Fusion was invented by the sun (at least in this solar system -- there are
theories that it was independently invented elsewhere too)!

~~~
zzalpha
Pfft, the sun just cribbed that idea from the big bang. Typical, unoriginal
sun...

------
kauffj
In the debate over the continued utility of human labor in the face of
automation, I suspect gene editing (and/or embryo selection) is the most
ignored countervailing factor.

~~~
circlecrimson
So all these truckdrivers that are being replaced with self driving rigs are
going to get into biotech now?

~~~
valarauca1
Nope. Self driving cars are gonna be a huge problem. I don't think many people
realize that nearly 9% of the US Employed workforce has no skill other then
driving a vehicle.

~~~
livingparadox
What exactly is to stop them from learning a new trade?

~~~
pc86
Nothing except time and money, but that's a _lot_ of people. Even occurring
over a decade or more it's going to cause the wages of a lot of those trades
to plummet.

~~~
livingparadox
The specific instance here was drivers, who in this hypothetical, will be
outcompeted by self driving cars, and and naturally find themselves with more
time on their hands whether they want it or not.

As to money, there are a number of trades you can learn without spending
money. And there are also jobs where you get on-the-job training.

I'm not saying it won't be a problem for these people, but eventually, most if
not all will adjust to the new situation and find themselves in a new job.

