How Does Gene Therapy Work?

Scientists have promised that gene therapy will be the next big leap for medicine. It's a term that's tossed about regularly, but what is it exactly? Trace shows us how scientists can change your very genetic code.

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How does gene therapy work?
"Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein."

Gene therapy trial 'cures children'
"A disease which robs children of the ability to walk and talk has been cured by pioneering gene therapy to correct errors in their DNA, say doctors."

Gene therapy cures diabetic dogs
"Five diabetic beagles no longer needed insulin injections after being given two extra genes, with two of them still alive more than four years later."

Gene Therapy for Cancer: Questions and Answers
"Gene therapy is an experimental treatment that involves introducing genetic material into a person's cells to fight or prevent disease."

How does gene therapy work?
"Gene therapy is the addition of new genes to a patient's cells to replace missing or malfunctioning genes. Researchers typically do this using a virus to carry the genetic cargo into cells, because that's what viruses evolved to do with their own genetic material."

Gene therapy cures leukaemia in eight days
WITHIN just eight days of starting a novel gene therapy, David Aponte's "incurable" leukaemia had vanished. For four other patients, the same happened within eight weeks, although one later died from a blood clot unrelated to the treatment, and another after relapsing.

Cell Therapy Shows Promise for Acute Type of Leukemia
"A treatment that genetically alters a patient's own immune cells to fight cancer has, for the first time, produced remissions in adults with an acute leukemia that is usually lethal, researchers are reporting."

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[sabafahim]

Anything that saves lives must be promoted

[Neuralatrophy]

It carries the potential to rectify genetic disorders in adults too. It would require considerably more treatment and immune suppression during. One of the side effects is the patients developing an immunity to the vector. The corrections also probably wouldn't be passed on like they would for children.

[kolrawktheworld]

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

[yidazhou]

Thank you for posting this! It's helped so much for Biology

[Neuralatrophy]

Just did a little reading. The wiki about gene therapy says "... Any modifications and effects will be restricted to the individual patient only, and will not be inherited by the patient's offspring or later generations". You need germ line therapy, which is directly to egg and sperm, to make the traits heritable.
Another article tackles the odds of somatic gene therapy affecting the reproductive cells as a billion to one or less.

[stupidstickmen]

This guy is talking WAY too fast for me to grasp the information....

[jackconnor]

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

[shadow]

using this vid on my graduation project for my presentation my topic in gene therapy lol

[LarryPeteet]

How do we add or change a gene in ALL cells?

[Teencat]

.exactly... how would it not end well if people were good, considerate, nonprejudice and respectful?

[rahulpaul]

Sir is there any kind of skin treatment available to change complexion genetically?

[ddx]

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.

[kphamu]

What are the side effects of gene therapy? Thanks for posting the sources.

[EjPichay]

trace is my favorite DNews reporter :D

[RoScFan]

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;

[bernardbrennan]

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.

[evanalexander]

This is how "I Am Legend" started

[Xexorian]

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

[Angelmind]

When I hear gene therapy and predictions I always think "Gattaca" Then I realize how old I am....I will never go to space.

[KiteFlyingGuy]

I actually always taught implementation of gene therapy would be hard because you have to replace all the old cells with the fixed ones, well atleast that's how I see it, but I geuss once you add the new cells they will slowly replace the old cells if they live long enough.