How Does Gene Therapy Work?

Gene therapy has the potential to save
millions of lives if we can just figure out how to make it
work. Hey peeps, thanks for tuning in to Dnews. I’m Trace. Gene therapy sounds like a nice easy treatment right that’s
therapy. In some ways it is on the macro level, but in your cells
it’s a little bit invasive. In gene therapy, doctors are basically
hacking the DNA of a living human. Using genetically engineered
retroviruses called vectors, scientists infect human cells. The
retrovirus can be programmed to carry a gene or a little bit of DNA that will
overwrite the messed up mutation and make it work
properly. It was first tried on a young girl in 1990 and despite some early failures it has the potential to
revolutionize treatment of genetic disorders. The Journal of Science describes one of the
recent successes that gene therapists say was really exciting. A few children were
born with metachromatic leukodystrophy which causes a defective immune system and some brain
disorders and kids who have it usually don’t live past the age of five. Bone
marrow contains stem cells, the cells normally produce red blood cells but
they can be reprogrammed using gene therapy it’s a little risky, but it can work. Taking bone marrow from
these kids doctors were able to infect the cells with a retrovirus and replace
the stem cells mutated gene with the repaired gene. Then they re-injected that back into
the patient and the fixed cells multiplied and as of the time we filmed this, the
patients are all in good condition, and are heading to kindergarten at that
time that others with that disease can’t even speak. There maybe future side effects but they
seem pretty happy with the result at the moment I mean I would be. Science just helped some
kids! Whoo! It’s not just useful in children. Scientists
have also used gene therapy on dogs to cure them of Type 1 Diabetes with two
of their doggie patients still alive years later. The treatment involved
injecting two things into dogs’ muscles. One gene to send glucose and an enzyme
to dictate glucose absorption. Scientists don’t have to target our DNA, they can also use gene therapy
to target the DNA of cancer cells. It’s like they gave cancer cancer. You’ve seen
this before if you’ve been following Dnews. A protein called CD47 is like a
passport that tells your immune system not to attack a cell. Normally cancer produces a ton of CD47. Using gene therapy on the cancer,
scientist turned off that cell production and let the immune system
blow it out of the sky like a decloaked Klingon bird of prey Gene therapy is still in its infancy but
the promise of future cures for everything from cancer to genetic
disorders is pretty incredible And I for one cannot wait to see what
this brings. What do you think of gene therapy? How do you see it being used in the
future? Tell us your thoughts and keep coming back for Dnews every day!


  1. Math, Science, Biology…. Hell, we are Biological computers with the outdated "Conscious/Subconscious – operating system" in dire need of grasping and keeping up with the momentum of our current technology.
    Moore's law tells us that it will never slow down, nor will it discontinue! So, why not do the same with our own avatars that dictate our livelihood? Sounds logical to me!

  2. The possibility of future side effects seems minimal compared to how their lives would be WITHOUT the gene therapy (if they didn't die before age 5). It will be interesting to see how their bodies react over time.

  3. wait a second…. if we are using custom made viruses to fix our DNA, whats stopping them from creating a zombie virus and a vaccine just to artificially wipe out a good portion of the population?

  4. Gene therapy is the scientific version of all the mutants and messed-up DNA found so much in superhero lore; it is literally, and I am using literally in the correct way, a method to create real-life superheroes;

  5. depends on whether the vectors also act on germ cells; gene therapy is currently very localized so I wouldn't bet on it; the viruses or whatever vector is used is likely not to reach the testicles or ovaries unless specifically targeted; so the next generation would still have the disease;

    the flip side is, if a vector is found that is sufficiently effective to act SAFELY on the somatic cells of an adult, they would absolutely work on the zygote or embryo;

  6. gene therapy actually is already being used on embryos and such; it is far easier to work with cells in a Petri dish when adminstering the vector;

    ultimately, the challenge for gene therapy is to act on adult patients; using the vector on a few cells that would multiple and thus have a systemic effect is easy; administering the vector to a large enough number of cells in a fully grown adult to have the same effect is the real hurdle of gene therapy;

  7. Yep, you are. By watching videos on YouTube, you are a consumer. The folks over at DNews are awesome enough and caring enough to provide their videos for absolutely FREE…but they still have to pay wages of all of their staff members, transport them to different locations to talk to different people, fund the research and everything else that goes into making their informative videos. A 30-second ad in the beginning of a video is a f***ing easy, cheap way for us to show our appreciation to them

  8. messing with peoples genes really fucks them up. pussies! most are cowards.and they like normal comfortable real world shit where nothing ever changes. people need genetic therapy, we were born with false, lackluster and simple human genes of the past millenium, nothing advanced or special.

  9. But what about the Goddamned corrupt world????will you force these cures on the rich and elites,cos if no one does, they will use the medical technology to inoculate and sterilize the poor and masses.

  10. this sounds almost like artificial evolution and i dont mind that, i dont get why people say that being human is the greatest thing in the world, example xmen, they have more abilities than the average person, yet still they are called freaks, HA the mutants have superior DNA, whose the freak now >:)

  11. Finally. Our evolved bodies are full of biological compromises. If we are lucky we will learn how to design far better individuals to make up far better future generations. Embrace The Ubermench my friend, and don't look back.

  12. You had me amazed with the news of this incredible new potential superfix, then made that last comment about bird of prey, and i was sold. I love this channel. Quickfixes of science in little tasty bites. I will learn more about gene therapy. Sounds amazing

  13. but how do you actually program genes?  it's not like you can plug it into a computer and write a script…

    when i was younger, i could drink as much milk as i wanted but now on later days i've become lactose intolerant. Shouldn't it be possible to re-program my genes to start producing whatever protein or w/e that made it possible for me to drink milk?

  14. I was wonder has anyone here looked into LOS large organ syndrome; as I wonder if it was due to leaving trash in a DNA sequence after disrupting a gene meaning a synthesised protein will be bigger than it should be and the size is magnified by a cells replication cycle?

  15. it also has the potential to kill millions of people.
    if we could hack the body to fight disease we could also hack the body to get ill.

    take ISIS if they got this technology could they create a disease that attacks white people. white being mostly western, 
    a nazi could create a airborne disease that targets anyone who does not have blond hair blue eyes or are tall and white. 

  16. Gene therapy has the potential to do a lot of good.. and some not so good things; but all technology is like that.. the proper oversight has to be instated, and common sense has to be used.  Gene therapy is not something that is easy to do, therefore it will likely not be performed outside of very controlled lab and clinical environments in the near future.

  17. I think this is a very promising future where we could potentially have the ability to cure someone before they get sick it would be a world without diseases

  18. Using vectors to rewrite the genetic code. Right now the target is to correct disease. Later it will be used to give people blonde hair and blue eyes. Could it be used to mark an entire population with a genetic marker without them knowing to mark them as "defective"?

  19. If I ever got gene therapy, I wouldn't do it to cure myself of a disease. I would use it to grow taller, get inhuman strength and agility, and absolute fucking invincibility.

  20. Gene therapy will work to the utmost via the entire lack of the presence of that which is unnatural. Unnatural : The use of flesh, dairy, timber & alloy as shelter, sustenance & propulsion chemical. I am not an educated life form on this matter……….. -_- <3

  21. super regeneration, immortality, change my hair color, better skin, change my bone structure, make my skin like steel / superman, my eyeballs deflect bullets, thanks gene therapy see you in 10 thousand years going into my bear cave to sleep off the changes

  22. can this change the shape of hairs permanently by using DNA from another person to permanently change hair? if so would it cost millions of dollars or is it possible to be a test subject? I want longer hair

  23. Just been told my lad who has Franconia anaemia A can have gene therapy!! So it's a very scary thing to consider but can potentially cure him

  24. Im from the future (2017), they just cured baldness and gray hair in mice studies. By turning on (or off, I cant remember) the KROX20 gene. I hope human trails start soon.

  25. My dad died of motor neuron disease, That's where you get progressively disabled, your muscles waste away and until your respiratory system fails. It's horrific and there's absolutely nothing anybody can do about it at the moment.   In Ireland it's a real death sentence because our health service will not provide respirators. A human life isn't worth that much really, that's the reality.Recent research has demonstrated the genes that may cause this. So hopefully gene therapy could be effective.

  26. this is goddam amazing. I read a book ( not real ) about this and it really got me interested in this. it is called charisma by the way.

  27. the sad thing is that 5 of those kids ended up getting cancer because the treatment went wrong, 4 of them manged to beat the cancer but one ended up dying, imagine the parents "you have a child who will die at 5, oh no now they should live normally oh wait they now have cancer, sorry'

  28. I once had the idea of infecting cells with a virus to alter the cell programming. Unfortunately I'm only 20 something and don't yet have a degree in science or a lab to work on my attempt to stop the aging process and cure diseases. I can't afford college and I'm not eligible for student loans or grants. So if I do nothing else with my life, I hope to make education more accessible, and less costly.

  29. He was supposed explain how gene therapy works. Replace and reinjected is all he said. Loves to talk but info is useless.

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