Niall Cummins

Biology 1090
Kathryn Randazzo
10/31/16

A New Way to Fight

A gene-editing system that was developed in conjunction by Carnegie Mellon University
and Yale University was recently used to cure a genetic blood disorder in living mice via IV(1).
This new technology is much more efficient than other gene-editing technologies that are in use,
such as CRISPR. The new technology decreases unwanted gene mutations. This offers a new
approach to treating genetic diseases.
Using CRISPR, cells had to be removed from the patients body. Enzymes were then used
to slice the DNA, and then the DNA was edited and the cells were places back in the body. Using
this new method, peptide nucleic acids(PNAs) are sent into the bloodstream, where they then do
all the work. First they open up the double helix, and then they bind to the specific target site of
the gene that needs to be changed.
Danith Ly , the main pioneer of this technology at Carnegie Mellon University claims
their new method using PNAs has shown up to a 7 percent effectiveness in mice with beta
thalassemia, compared to CRISPRs 0.1 percent effectiveness.
The reporter at Science Daily states that, This new method may be used in the future to
treat people with diseases such as sickle cell anemia.(1) It may also be used to treat other
genetic disorders. The true scope of effectiveness of this technology is yet to be seen, as it is very
new.
Some claim that a 7 percent effectiveness rate is not enough, however, it is enough to
change the phenotype in mice to show that they no longer had beta thalassemia. The mice that
were treated are now producing hemoglobin at a normal rate. Some also say that this may not be
the most effective way of treating these diseases because the treated mice had to receive a
treatment every few months for the effects to last. I think it is still effective even if they do have
to receive lifelong treatment. Receiving an intravenous treatment every few months is something
I am sure many sickle cell anemia patients would love.
The scientists mentioned in this article are all students or graduates at Carnegie Mellon
University. I highly doubt that they have altered their conclusions, as they are at a very
prestigious college in the scientific community, and I dont see what motive they would have to
lie about their findings.
Before I read this article, the only information I knew about gene-editing was what we
have learned so far in this class about GMOs. I had no idea that a system such as CRISPR was
already in place, treating people, and I had no idea that our technology was anywhere close to

being able to treat people for genetic disorders intravenously. I find what I read in this article to
be very fascinating, and I hope to one day see this technology in use in the real world, helping
people.
I think that the person who wrote this article made it very easy to understand how the
complicated technology involved works. They did a great job of explaining the systematic
procedures of both CRISPR and this new, unnamed technology. To back up my claim, here is an
excerpt from the article explaining the differences in these two systems:
Gene-editing technologies like CRISPR rely on DNA-cutting enzymes to slice open DNA at a target site
to edit a specific gene. The problem with this is twofold. First, the enzymes are large and therefore difficult to
administer directly to living animals, so scientists typically remove the cells, treat them in the lab and then put them
back into the body. Second, once inside a cell, the enzymes could indiscriminately cut DNA at sites other than the
original gene target.
The new Carnegie Mellon/Yale system avoids both of these issues. The new system consists of
biocompatible nanoparticles containing PNAs, small nano-sized synthetic molecules in which a protein-like
backbone is combined with the nucleobases found in DNA and RNA. PNA is designed to open up double-stranded
DNA and bind near the target site in a highly specific manner without cutting anything. And the PNAs easily fit
inside the nanoparticle delivery system, which is FDA-approved and has already been used to treat
neurodegenerative diseases in humans.(1).

Im excited to see this technology leave the ground in the coming years, and I am even
more excited to see what innovations happen next in this industry.

Citations
1. Carnegie Mellon University. "New gene-editing technology successfully cures a genetic blood
disorder in mice: Technology may offer a minimally invasive treatment for genetic disorders of
the blood." ScienceDaily. ScienceDaily, 26 October 2016.
www.sciencedaily.com/releases/2016/10/161026110626.htm
Jocelyn Duffy.