CRISPR-Cas9 is a gene-editing
technology that has been continually improved since its creation. This
technology can be used to prevent and treat various genetic diseases. Recently,
scientists have developed a new and effective way to deliver this technology
inside cells. By using gold particles to deliver new genetic material to a
cell, these scientists have modified and improved the CRISPR-Cas9 mechanism.

            Today, CRISPR-Cas9 is the simplest
and most precise method of gene editing that is available to medical
researchers and geneticists. In order for CRISPR to modify DNA, Cas9, an enzyme
that cuts the DNA at a certain location, donor DNA and a piece of guide RNA
(gRNA) are needed. The gRNA locates and binds to a certain sequence within the
DNA and guides the Cas9 enzyme to that section. Once in the proper section of
DNA, the Cas9 then cuts both strands of the double stranded DNA in the correct
place. This causes the cell to begin to repair the DNA that has just been
damaged through homology-directed DNA repair. The cell uses the donor DNA that
is provided to it and mends the DNA.

            There are problems with CRISPR-Cas9 in
regard to its safe delivery of the essentials for gene editing into the cell. CRISPR-Cas9
often uses viruses to deliver genetic material into a cell. This has obvious
complications and risks that come along with it. One example is the use of
retroviruses as vectors to deliver genetic information into the T-cells of a
patient in order to kill cancer. This is a problem because if the genetic
information is not properly delivered, that patient’s cells will attack each
other and the patient will die. It has often been noted that the mistakes that
are made by CRISPR-Cas9 are results of a delivery malfunction.

            In order to fix the delivery issue,
researchers at the University of California, Berkeley, have created a vessel
for the necessary genetic information. CRISPR-Gold, as it has been cleverly
named, does not utilize viruses but instead uses gold particles to transport
Cas9, gRNA, and donor DNA into the living cells of an organism. The gold
particles that form the vessel bind all of the gene-editing components together
and, once inside whatever cell has been targeted, releases them in order to
trigger homology directed repair. Once injected into an organism, the cells of
that organism recognize a marker within the CRISPR-gold and transport the
delivery receptacle into the cell where it releases the Cas9 enzyme and the
donor DNA.

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            In the study that Niren Murthy and
Irina Conboy performed, the CRISPR-Gold mechanism’s performance was evaluated
in mice with Duchenne muscular dystrophy. A single injection of this
CRISPR-Gold technology restored 5.4% of the gene that codes for the disease to
the wildtype sequence. The mice treated with the Cas9 and donor DNA without the
gold particle shield only experiences a 0.3% correction rate. Comparatively, it
is clear that the gold particle vessel makes the CRISPR technology much more
effective. In fact, within the study on the mice with muscular dystrophy, the
CRISPR-Gold mechanism had a correction rate that was 18 times higher than the
trial without the gold particles.

            In conclusion, the CRISPR-Cas9
gene-editing technology has been constantly improving since its initial debut.
The most recent advancement, a vessel made from gold particles, has been to be
a safer and more effective method of transporting genetic information into a
target cell than the common method of using a virus. With this new advancement,
this technology is one step closer to becoming a common, effective and safe way
of treating genetic diseases within the human population.



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