What if you or someone you loved was born
an inherited disorder and you were told it
could be cured by a relatively simple treatment
that included genetically correcting your
DNA?!
Would you do it?
Hey gene-ees, it’s Trace for DNews.
With new gene editing technology emerging
from Carnegie Mellon and Yale Universities,
genetic engineering may be up in us sooner
than you think...
Researchers at Carnegie Mellon's Center for
Nucleic Acids Science and Technology (say
that 10 times fast!) have developed a way
to treat inherited blood disorders using synthetic
molecules called peptide nucleic acid molecules,
or PNA.
PNA is a biomolecular structure created by
researchers that can mimic DNA as it is structured
similarly.
Think of PNA as a blank document that scientists
add a genetic code to.
PNA can be designed to bind to specific parts
of a gene sequence, which allows scientists
to fix a malfunctioning piece of DNA that
is the cause of a disease.
In this particular case, they are fixing the
gene that is responsible for a blood disorder.
So, in this new study, scientists coded the
PNA to target haematopoietic stem cells that
make other blood cells.
They then injected it into a living organism.
The PNA slipped into the cells, found the
site of the problem, and then bound itself
to the malfunctioning DNA to form a PNA-DNA-PNA
triplex chain, ‘mending’ it by correcting
the sequence.
Right now, this technique is used to treat
inherited blood disorders.
According to the World Health Organization,
nearly 5% of the world is born with inherited
hemoglobin disorder, like sickle cell anemia
and Beta thalassemia.
Beta thalassemia disrupts the creation of
hemoglobin and causes extreme anemia, stunted
growth, and enlarged organs, among other ailments.
Until now, there had been no real ‘cure’
for Beta Thalassemia, but this new gene editing
technique may be the answer.
In mice, researchers have seen a 7% success
rate in curing Beta thalassemia.
This may sound low, but other gene editing
treatments have only been .1% successful.
Also, in a disorder like Beta thalassemia
a 100% cure is not necessary to regain improved
health.
Even if the person isn’t cured, they may
be able to produce more hemoglobin to help
alleviate their condition.
The FDA has approved this synthetic nucleotide
technology after testing it on mouse and human
bone marrow.
The technique has advantages over the other
gene editing treatment you may have heard
of, CRISPR-Cas9, in two ways.
First, CRISPR cannot simply be injected into
the body like this new PNA gene with the correcting
process.
With CRISPR, cells have to be removed to receive
treatment in the lab and then they are injected
back into the body.
Second, with CRISPR, once the cells are in
the body, there is a chance that the enzymes
will indiscriminately cut the DNA at the wrong
place, thus rendering the treatment useless
and it could be potentially harmful.
This does not happen in this new treatment.
The technology has excited scientists as it
can possibly lead to developments in curing
other genetically inherited blood disorders,
like sickle cell diseases that affect 100,000
Americans.
And hopefully other diseases in the not-so-distant-future!
If you’re interested in learning about diseases,
maybe you like the Animal Planet show Monsters
Inside Me; has great computer animation.
Now you can watch that and other Animal Planet
shows on the Animal Planet Go app.
Download for free at your app store or check
out the link below.
If you’d like to know more about CRISPR
and how that also has huge promise, check
out THIS episode.
What do you think?
Would you be willing to try this new gene
editing technique to fix an inherited disorder
or disease?
Tell us in the comments below, then subscribe
to get more DNews every day.
