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!
