Editing Genes Inside the Human Body


[♩INTRO] Last week was a pretty big deal for the future
of medicine. For the first time ever, researchers injected
someone with tools that would edit his DNA, hopefully curing his genetic disease. If it works, it’ll be a huge step toward
treating some of the world’s most debilitating genetic diseases. But despite the connections being made in
some places, this isn’t ushering in the age of designer babies. On November 13th, a man named Brian Madeux
received billions of carefully-designed, gene-editing viruses via an IV. Madeux has Hunter syndrome, which means he’s
missing an essential enzyme for breaking down certain kinds of large sugar molecules. Instead, these sugars accumulate in his cells,
causing them to swell, and making his tissues and organs grow to abnormal sizes. Hunter syndrome can lead to all sorts of problems,
including less mobility, vision and hearing loss, brain degeneration, and heart disease. And like many rare genetic diseases, it has
no cure. Patients can get enzyme boosts to ease their
symptoms, but they’re incredibly expensive, and the enzyme levels drop off quickly. Gene editing — if it works — would be
a much more effective treatment. The idea is almost exactly what it sounds
like: you take a patient’s genes, and you edit them, usually with the goal of fixing
a mutation or curing a disease. Researchers have been trying to cure diseases
this way since 1989, but in the last few years, gene editing techniques have gotten much more
accurate and reliable. There are already some approved treatments
that involve taking the patient’s cells out of their body, modifying their genes,
then putting the cells back in. With Madeux, researchers are doing something
new: the tools to edit his DNA were injected directly into his blood to then go modify
cells elsewhere in his body — in this case, his liver. While most of the gene editing research you
hear about these days uses a tool called CRISPR, this treatment uses a system known as zinc-finger
nucleases. It’s a slightly older method that’s harder
to design and make, but it also has a good track record. Zinc-finger nucleases consist of two essential
parts—a DNA-clipping enzyme, and a section that binds directly with the part of the genome
you want to cut. To edit genes, scientists design a pair of
these DNA clippers that cut both halves of the DNA strand. If the bit of DNA they want to add is designed
to match the sequences at those broken ends, then when the cells go to repair the break,
they’ll sometimes include the new gene. For Madeux’s treatment, the blueprints for
these gene-editing tools were packaged in modified viruses, which, you know, infect
cells — the way that viruses do. Then, all the team needed to do was put them
in a plain old IV. If all goes according to plan, Madeux’s
liver will transform into a mini manufacturing plant for the enzyme he lacks. According to the researchers, if the gene
gets into 1% of his liver cells successfully, he’ll make enough of the enzyme to treat
his condition. But that’s a big if. Gene therapy trials are known to be risky. People have died when the viruses used in
other types of gene therapy treatments sent their immune systems into overdrive. And that’s not the only potential complication. Sometimes the introduced genes land in the
wrong spots and cause cancer. And if the gene is activated in the wrong
cell type, it can cause other issues. The new treatment is designed to avoid putting
the gene in the wrong place very often, and there’s a failsafe that’s supposed to
prevent the insert from working in any non-liver cells. But there’s always a chance something could
go wrong, and if it does, there’s no going back. They won’t be able to un-edit his cells. That’s the risk—and reward—of what Madeux
signed up for, and it may take several months before we know whether the experiment succeeded. Even if things do go smoothly, though, the
therapy isn’t necessarily going to cure him completely or indefinitely. This genetic editing should help with most
of the symptoms, but the enzyme probably won’t be able to cross the specialized barrier that
protects the brain, which means it won’t be able to prevent brain damage. It also can’t reverse the damage the disease
has already caused over the past 44 years of his life. And the therapy should work for at least several
years, but how long it remains effective depends on the age and type of cells that incorporate
the new gene. The ultimate goal, though, isn’t to treat
adults like Madeux and the other volunteers in this study—it’s to develop a safe enough
protocol to use in children or even infants. That way, they’d be spared the syndrome’s
worst effects. And if this treatment works, researchers will
also be a lot closer to developing similar gene therapies for other types of diseases. But one thing this research will not get us
closer to is some dystopian future where we’re genetically engineering our children to have
extra fingers or whatever. Whenever there’s a new development in gene
therapy, there’s a lot of talk about the risks of babies with edited genes, or how
to draw the line between editing an embryo’s genes to eliminate a disease and custom-designing
a person. And those are real ethical problems that we’ll
need to solve before we start using gene editing to change someone’s genome before they’re
born. But research like this, where scientists are
trying to treat a disease in a living person, is totally different. The changes to Madeux’s DNA won’t be happening
in any of the cells destined to become sperm, so there’s no way for him to pass them on. This is a pretty big leap for gene therapy,
especially if it works without serious complications. But it’s not designing babies. Alright, I’ve got an EXCITING ANNOUNCEMENT
for you. The SciShow team and I were talking about
a common problem that many of us have. When people ask us what we want for Christmas
or for our birthdays, often, we won’t know what to say and then they’ll get frustrated
and we’ll get frustrated and it’s like “I just wanted to do a nice thing for you
and you’re being so stubborn!” But I don’t know! I don’t care about stuff that much. But I was thinking, you know, there are a
few things that I would like to get or that I’ve bought for myself of that people have
gotten me that I loved because I love the world and the universe and understanding things
and the existence of life and doing experiments on my proteins — that kind of thing. So, we’ve put together a collection of artifacts
of this universe. We got a limited number of each of these things
and we’ve put up at a store called SciShowFinds. These SciShow Finds are curated by me, they
are things that I know I would love to get in my stocking. It’s a very small list, just six cool things
with varying price points. And I did my best to only include one science
book, It was very hard to pick, but I picked the one that had the most impact on me of
all the books that I read this year. It’s called “What is Life” and it makes an
extremely compelling case that biology is like a chemical inevitability. And, along the way, you learn a whole lot
about chemistry, and it’s great, because — you might end up googling things every
once in awhile to make sure you know what the author’s talking about — but at the
end of the book, you will understand chemistry in a whole new way. We’re probably going to add new finds…as
we find them…throughout the year, and the new ones will replace these old ones, so these
products are only around for a limited time. You’re bound to have friends or family who
would love these Mars Socks, trilobite fossils, or this Space Shuttle lapel pin. And, if not, you might want to get them for
yourself or just send that link over to somebody who’s saying to you, “what do you want
for Christmas?” and it’s like, “Just anything from this website, Ma. You’ll be getting me socks that I’ll actually
appreciate this time.” And know that when you buy from SciShowFinds.com,
or you send that link to somebody else, you are also supporting SciShow. So, thank you for doing that. And I know what you’re thinking, “Can
I get that cool SciShow hoodie at SciShowFinds.com? You can’t. You can’t. This is just for staff. Sorry. [♩OUTRO]

100 thoughts on “Editing Genes Inside the Human Body”

  1. Can someone explain to me what is wrong with "designer babies"? The way I see it you end up making near perfect humans from the start, largely removing any negative conditions someone can get over their lifetime because they lost the genetic lottery. I highly doubt parents would think it would be a good idea to give their kd green skin or extra arms or something.

  2. LOVE the scishow finds idea!! It would be great if you tell us when there is something new to see there! Also-thanks for covering this topic, and making the info complete yet conscise! I tried reading an article about it, and it was just too much to get through for me-this was perfect! I sincerely hope it works well for this guy so that it can help others!

  3. Hmm thought this was going to be about this equally impressive recent development where they used gene editing to correct skin cells then grow those skin cells and grafted them back on: https://www.theatlantic.com/science/archive/2017/11/epidermolysis-bullosa-new-skin/545291/ . Although this takes it a step-further and injected them which is also amazing.

  4. Wasn’t this how the movie Legend (Will Smith) started? A scientist creates a virus that can target and kill the cancer cells, but that virus mutates to become the viruses that zombify people. Yes, zombify is a word. 🙂

  5. Ok, so if the genes cannot be passed on? Doesn't that mean that his offspring has a chance of developing the same issues? Seems as if it should be the responsibility of the researchers to find a more genetically permanent answer to these issues or else this treatment is more about exploiting people pockets for a genetically temporary answer to the issue. I will say, at least the quality of the lives of the individuals will increase, but it seems like a ploy ( not that it doesn't work, just that its seems as if there could be more permanent ways of taking care of that. Maybe the treatment SHOULD have a gene therapy option to pass those gene edits to the sperm cells (or what ever) for a more permanent solution for the peoples buck. Its funny how buisnesses, and corporations ask thier employees for permanent solutioname, but the businesses NEVER offer permanent solutions. No MONEY to be made there. I swear the world is run by greedy ass wholes, who really think money can buy everything. It shows without having to be spoken. All you have to do is open your eyes and look. Money = False Power.

  6. If any man shall add unto these things, God shall add unto him the plagues that are written in this book:
    19 And if any man shall take away from the words of the book of this prophecy, God shall take away his part out of the book of life, and out of the holy city, and from the things which are written in this book. Your Call.

  7. I don’t see the problem with designer babies… You just need to have the social failsafes in place to prevent a eugenics friendly and favoritism scenario. Its the same dilemma if we encounter aliens with differing abilities than us or if we make robots that are physically and or mentally superior… We just need to have society remain stable and keep equality even if there are inequalities… failsafes…

  8. This is terrifying news, how long until someone can poison the water and mass alter a population without us even knowing?

  9. we're worried about the ethics of building a baby that can better handle the world and yet circumcision is still a thing

  10. I'm all for preventing disease and complications in babies… but y'all got some of those permanent CRISPR benefits for us adults?

  11. Hello @SciShow, can you please do a SciShow edition video about the head transplant surgery that will happen this following month.

  12. Wait… first time? What about the little girl that went through gene therapy like over a decade ago using bacterial payloads to edit her genes?

  13. Im sorry, how is this not going to usher in designer babies? This is the first possibly successful gene correction. I don't see why the gene for blue eyes are different to the genes for hunters. Sure, its an infant/embryo vs adult, and yes, the genes will be existing in many more cells than just a target, but this looks like a high profile case that opens up the field. I would love to see your side, but without it I feel this is one more step on the path to demihumans and superhumans.

  14. Wouldn't it be better if the gene therapy affected his sperm? That way he can't pass on his debilitating disease to children, right? You said that it won't affect his sperm like it's a good thing, but it's a tragedy waiting to happen.

  15. Goodness, it's like what Louis Pasteur did with that one kid who was infected with rabies.

    At least, I hope it ends up being like that. I'm not the best at being happy for other people, but, if this works, it'll be a huge step forward for progress in general.

  16. Couldn’t this cause someone with bad intentions to edit someone’s genetics to give them a new disease or something?

  17. Genetic diseases like Huntington’s, hemophilia, and sickle cell unfortunately won’t go away naturally. the only ways to cure diseases like this are genetic engineering or eugenics, and I think we ALL would prefer engineering.

  18. If this stuff works (and epigenetic treatments too), then CF, Down's, Rhett and other nondisjunction and mutation disorders can truly be cured.  Other non-genetic disorders, like autism, could benefit too.  Still, IF.

    And if it fails et al, well, that's how nature works.  Nature finds a way.  Symbiotic treatments will work far better than traditional 'kill it, change it' treatments of today.

  19. I'm a sophomore in high school and have been researching things dealing with gene therapy because its so cool and advanced. I recently met a doctor and talked to her about the ups and downs of her researching gene therapy for children with this disease where they have no backbone,,,,,anyways her first patient for gene therapy trials is in 2018 and I'm hoping everything goes well.

  20. lel chemistry in biology say whaaat how else would you explain photosynthesis or cellular respiration that catabolizes 2 pyruvites into acetyl CoA enzyme that goes through a redox process and generates ATP along an ongoing Krebs cycle and how the plant counterparts has photsytem 2 and 1 and then the calvin cycle that uses Rubisco to finalize photosynthesis in both CAM C3 and C2 plants

  21. Hello American friends, I live in Brazil and wanted someone to train my English. For I study this wonderful language English. Were you interested? My Facebook: João Paulo Fillho

  22. The Healthspan Discord community discord.gg/ftSbffu discusses this and other topics in biotech / regenerative medicine, and how aging is currently being cured, come and say hi!

  23. Dang it Hank, Dave is so much better at selling than you are. When we want the hoodie – which we do, of course! – you say… "Well, for this exclusive hoodie, you'll have to go over to DFTBA Records and buy one for an especially high price… like $100," – so people will be all, "Dang, man! That's something like Dave would say! I have to get myself and everyone I know that exclusive hoodie!"

  24. Not about the video of course, but if offered, I would absolutely design my baby.
    By the way, I really hope the gene editing works for this guy.

  25. Read this!

    I fear if they use it on children or infants, it's not like doctors and teachers don't already say that kids have conditions that they don't. Just think about it!!! Their would just be a bunch of poor little children ZOMBIES walking around, never knowing what it's like to be a kid. Messing with genetics is already dangerous enough, but if they DO plan on doing this with children and infants -lets just say I don't think any humans worth living would live long enough to know the the consequence.
    Do you want to live in a world even worst then the giver?

  26. Give credit to scientists not to doctors….a Doctor would have just handed over the patient a check of sum to cure the disease with 80% profit….Scientist doesn't have a job they think…think something which is not in book of knowledge….

  27. I don't know about the semantics you're using here. I agree that there's a difference between this and designer babies but I don't think the difference has anything to do with when the genes are edited or whether they are passed on to future generations. You state in the video that the goal is to edit the genes of children or babies so why would it make a difference if it was done 2 months after birth or 2 months before? And wouldn't making the edited genes inheritable be a good thing (so future generations can avoid this expensive and potentially dangerous process)?

  28. I have a genetic disorder which technically has a treatment. No one's ever purchased it because it costs $125,000 per injection (bit costly to make).

    You get one injection (into your spine) every two months or so and it pretty much halts your disease in its tracks. It's slowly making me weaker and I'm really losing the ability to talk/swallow/breathe. I think if it wasn't affecting my speech, I probably wouldn't even know I had it. I'll probably be mute in a couple more years. I can live with that since I'm also a writer 🙂

    Mostly infants get this and they die in a year or two, so if I could afford it? I'd donate it to a family who was watching their child die because they couldn't afford the treatment. It's not expensive because "Big Pharma" is greedy. It's expensive because it's monstrously hard to make and takes loads of Ph.D.s to generate even small quantities of it. They're definitely looking for cheaper ways to make this stuff, but it isn't easy.

  29. Hah! That's all I wanted (the hoodie)! And thanks for this vid! I'm sharing this with my bio-chem professor and his colleagues along with some related articles on this research! Hopefully he'll be another subscriber!

  30. There are over 200 requirements for Life as We Know It On Earth to exist lots of small things that you wouldn't even think of affect things a lot more than you would ever realize from the surface take a look deeper and you'll realize things aren't exactly the way we perceive them to be

  31. I stumbled across an interesting blog post on the topic of editing genes. If you liked this video, you might want to take a look at that blog for commentary. https://dailyflabbergast.wordpress.com/2017/11/20/our-body-is-our-identity/

  32. Future question: Why is it that when men get over the age of about 60 they start to give off a constant bad smell no necessarily connected with farts or bad breath? I heard it once described as "men hit a certain age and then start rotting from the inside".

  33. it bugs me that people think gene editing is a bad thing or an ethical dilemma i feel this is a very black and white issue if you can remove the risk of something like alzheimer's or get rid of a genetic disease who cares if some rich guy wants there kid to have blonde hair and blue eyes

  34. Are SciShow videos considered "scholarly"? I'd like to use this video as a source for my research paper on gene editing, but we're only allowed to use scholarly and peer-reviewed sources.

    Either way, thanks to the Sci Show team!

  35. 4:39 well that line, right there, was pretty obviously set
    one, is helping a human have a normal life, another is steroid boosting all their "stats" from second 1 (stats just as a joke, as in stronger, more endurance, generally smarter, stuff like that)

  36. Why do you think it would be dystopian to optimise the genes of your children ?
    If there is any way, I could make the DNA of my children more suitable for them living a healthy and productive live, I would pay as much as I can afford to do so !!!

  37. I hope these technologies keep advancing at such a fast pace. I'vr got a genetic disease myself, and it would be pretty nice to get rid of.

  38. Totally in favor of gene editing for embryos when the techniques become reliable. That development will change public opinion on eugenics from a dirty word to a fashionable and progressive practice. Creating a new generation free from disease and pathology is an objectively ethical and moral good.

  39. how did this name happen??
    when i think of someone with low mobillity, weak perception, degraded brain and a weak heart a hunter dosent come to mind

  40. I mind gene editing experiment has been increased since it was 1989year because my niece died in 2007year. She was 10years old. Her brain blood pipe suddenly rupture.

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