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!