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  • 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!

Gene therapy has the potential to save millions of lives

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How Does Gene Therapy Work?

  • 77 11
    James posted on 2015/06/17
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