Engineers Made Fake DNA To Fix Genetic Disorders
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Engineers Made Fake DNA To Fix Genetic Disorders

What if you or someone you loved was born
an inherited disorder and you were told it could be cured by a relatively simple treatment
that included genetically correcting your DNA?! Would you do it? Hey gene-ees, it’s Trace for DNews. With new gene editing technology emerging
from Carnegie Mellon and Yale Universities, genetic engineering may be up in us sooner
than you think… Researchers at Carnegie Mellon’s Center for
Nucleic Acids Science and Technology (say that 10 times fast!) have developed a way
to treat inherited blood disorders using synthetic molecules called peptide nucleic acid molecules,
or PNA. PNA is a biomolecular structure created by
researchers that can mimic DNA as it is structured similarly. Think of PNA as a blank document that scientists
add a genetic code to. PNA can be designed to bind to specific parts
of a gene sequence, which allows scientists to fix a malfunctioning piece of DNA that
is the cause of a disease. In this particular case, they are fixing the
gene that is responsible for a blood disorder. So, in this new study, scientists coded the
PNA to target haematopoietic stem cells that make other blood cells. They then injected it into a living organism. The PNA slipped into the cells, found the
site of the problem, and then bound itself to the malfunctioning DNA to form a PNA-DNA-PNA
triplex chain, ‘mending’ it by correcting the sequence. Right now, this technique is used to treat
inherited blood disorders. According to the World Health Organization,
nearly 5% of the world is born with inherited hemoglobin disorder, like sickle cell anemia
and Beta thalassemia. Beta thalassemia disrupts the creation of
hemoglobin and causes extreme anemia, stunted growth, and enlarged organs, among other ailments. Until now, there had been no real ‘cure’
for Beta Thalassemia, but this new gene editing technique may be the answer. In mice, researchers have seen a 7% success
rate in curing Beta thalassemia. This may sound low, but other gene editing
treatments have only been .1% successful. Also, in a disorder like Beta thalassemia
a 100% cure is not necessary to regain improved health. Even if the person isn’t cured, they may
be able to produce more hemoglobin to help alleviate their condition. The FDA has approved this synthetic nucleotide
technology after testing it on mouse and human bone marrow. The technique has advantages over the other
gene editing treatment you may have heard of, CRISPR-Cas9, in two ways. First, CRISPR cannot simply be injected into
the body like this new PNA gene with the correcting process. With CRISPR, cells have to be removed to receive
treatment in the lab and then they are injected back into the body. Second, with CRISPR, once the cells are in
the body, there is a chance that the enzymes will indiscriminately cut the DNA at the wrong
place, thus rendering the treatment useless and it could be potentially harmful. This does not happen in this new treatment. The technology has excited scientists as it
can possibly lead to developments in curing other genetically inherited blood disorders,
like sickle cell diseases that affect 100,000 Americans. And hopefully other diseases in the not-so-distant-future! If you’re interested in learning about diseases,
maybe you like the Animal Planet show Monsters Inside Me; has great computer animation. Now you can watch that and other Animal Planet
shows on the Animal Planet Go app. Download for free at your app store or check
out the link below. If you’d like to know more about CRISPR
and how that also has huge promise, check out THIS episode. What do you think? Would you be willing to try this new gene
editing technique to fix an inherited disorder or disease? Tell us in the comments below, then subscribe
to get more DNews every day.

100 thoughts on “Engineers Made Fake DNA To Fix Genetic Disorders

  1. Why Do we feel cold when we feel sad…. Or when we get scared our body temp reduce why is that so…? ⚠⚠⚠⚠⚠⚠⚠⚠⚠⚠⚠⚠⚠⚠⚠⚠⚠⚠⚠⚠⚠⚠⚠⚠⚠✔✅✔✅✔✅

  2. The only way to advance as a human race and allow these cures and treatments to happen, we need to destroy the pharmaceutical companies, because they keep us sick.

  3. I'd try it eyes closed. I have cystic fibrosis so… I'm counting on that kind of new treatments so I don't die ! Thanks DNews, you're awesome !

  4. wod it be possible to make a spaceship that can go hiperspece that can go to a new universe before the end of the universe.

  5. Couldn't all of those bindings to DNA cause some form of deterioration or shorter life in return for curing the disease? And if so how does that affect offspring or will it only affect the subject treated upon.

  6. "What do you think? Would you be willing to try this new gene-editing technique?"
    If I can afford it, and once the bugs are worked out, we'll talk.

  7. Ahhh!! this is very exciting! Talk about a platform for curing so many symptoms. This in combination with the obvious stem cells!! ohh baby i'll be a cyborg by 55 you bet, and i'll probably be buying a new liver at that point too 😉

    probably 35 going to need that liver :/



  8. So the ubermensch will come, not only the newborn also the senior will return to youth and will become at least 500 years old. As long u can afford that treatment.

  9. will it ever be able to cure dyslexia I'm guessing not but oh my god would I love to be able to read properly I wonder if science will ever be able to fix that

  10. What would happen if you edited your DNA to give yourself different colored eyes, or larger genitals, or a different shaped nose. Would it be painful, would it have no effect, or would there be a seamless transition?

  11. Hell YES. I'd do that in a heartbeat. There's reasons we can do this stuff and that's to HEAL and CURE people. God botherers be damned.

  12. Unequivocally the answer is yes. We're just working on the organic machine… and I'd feel much more at ease with a PNA treatment than with a lug-wrench.

  13. Been hearing about things like this since I was born (with severe Hemophilia A from mutation), but none of them panned out until now. This seems incredibly promising, and I'd sign up in a heartbeat as long as there wasn't reason to doubt its efficacy or think there would be significant risk of complication.

  14. Considering how many people should have died due to ailments or diseases to root out bad gene pools I feel worried about where medical science is propelling us in the future. There is a bitter pill to swallow when loved ones or yourself need treatment to live so medical help is natural but then when looking at how dependent we have become it just seems more and more that some things are meant to be for a reason.

  15. The presenter here is like the happier and cheerful version of the presenter in the Seeker who is more serious and somber.

  16. how would gene therapy work? would a single solitary altered DNA strand take over the entire host? if so.. the weaponized capabilities of this would be unimaginable.

  17. I do wonder if it is genetically temporary. If it gets passed on genetically, I do wonder what the effects are on the later generations.

  18. I don't mind this, as long as we aren't going overboard and leading ourselves to genetic conformity. Anybody trying to spread terror through disease would REALLY love genetic conformity, because that's less variety to account for.

    Not that humans are all that diverse to begin with. We're like a Windows operating system.

  19. I wonder what they mean as fixed. Fixed as in you the patient won't have that defect anymore or fixed as in your future offspring won't have that genetic disorder or possibly the human gene pool as a whole. What if the fix is good for a few generations and after that it reverts back to it's original state before being fixed? Could this lead to a catastrophic gene pool corruption?

  20. I am open to this technologie (I know several people, who would oppose it as "playing God"), but as long as sucess rate isn't above at least 50% I wouldn't want this treatment on humans (maybe grown up volunteers, who had agreed before hand despide knowing the risks).

  21. Early on, Trace asked a question, to wit: if you had an inherited disorder and it could be cured with a relatively simple treatment that included correcting your DNA, would you do it?

    I have a resounding answer to that: YES! Because I have such a disorder, and I'd give anything if it were cured. It's an inherited disorder that my grandmother, my cousin, and my nephew also have; and my grandmother said that an aunt or cousin of hers had it, too. It has ruined much of my life, and I would give anything to be free of it and its ill effects.

    But I'm almost 60 years old. I bet that by the time a DNA-correcting cure becomes viable, I'll be almost 70. Society, i.e., CMS* and the insurance companies, will run a cost-benefit analysis and determine that at such an advanced age, it won't be worth the monetary outlay to treat someone of such advanced years. So I and others in my age group will be declared ineligible.

    I expect that such a cure will come along, probably in the next few years. But people of my generation won't be eligible, and there is nothing I or anyone else will be able to do about that.

    * That's the Centers for Medicare and Medicaid Services (U.S.), or whatever succeeds it.

  22. Any chance that they can start creating genes for an adeptus astartes primarch? Would be nice to see one of those while I'm still alive.

  23. Okay sounds great. Some people will be idiots and say this is bad and come up with weirds ass reasons but from what i saw this may just be what is needed to be done…like it or not everything we are exposed to changes our DNA. So whats wrong with trying to fix it 💁‍♂️

  24. only a matter of time before this treatment gets slurped up by big drug companies, never to be seen again

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