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