May 2024 SOCRA Source Journal - Journal - Page 56
The Potential of Genome
Editing: A Tool to Rewrite the
Aging Story
DNA, or deoxyribonucleic acid,
is like a blueprint for all living
things. It’s a long molecule that
contains our unique genetic
code, which guides how we
grow, develop, and function.
As we get older, our DNA can
get damaged or start to wear
out, just like parts in a machine.
This damage can cause our cells
to work less ef昀椀ciently or stop
working altogether. Over time,
this leads to the physical signs of
aging, like wrinkles or gray hair,
and can also increase our risk of
age-related diseases like heart
disease or Alzheimer’s. So, in a
way, our DNA holds the story
of our aging process, and by
understanding it better, we might
be able to change that story.
Genome editing is like an
advanced spell-check for our
DNA. It’s a technology that
allows scientists to make precise
changes to speci昀椀c parts of our
DNA. Imagine being able to
昀椀nd a typo in the instructions for
building a part of our body and
being able to correct it. That’s
what genome editing can do.
Now, when it comes to reversing
aging, genome editing has a
lot of potential. Remember the
DNA damage that contributes
to aging? Well, with genome
editing, we might be able to
昀椀x that damage, sort of like
repairing the worn-out parts in
a machine. This could help our
cells work better and for longer,
potentially slowing down or even
reversing the aging process.
So, genome editing could be
a powerful tool in our quest to
understand and control aging.
56
Promising Advancements
One notable example of the
potential of gene editing in age
reversal is a study from Harvard
Medical School, where scientists
used gene-editing tools to treat
vision loss in mice1. The mice
had a condition similar to retinitis
pigmentosa, a common cause
of blindness in humans. The
scientists targeted a mutation in
the enzyme PDE6βK, a protein
crucial for visual signaling in
the retina. Using gene editing,
they were able to correct this
gene mutation, restoring the
enzyme’s activity in the mice’s
retinas, preventing the death of
photoreceptors, and restoring
normal electrical responses to
light. The mice, after undergoing
gene editing, exhibited behavior
typical for a mouse with healthy
vision, and their improved vision
persisted into old age.
In another study, researchers
at the Karolinska Institutet and
the Institute for Basic Science
in South Korea present a
compelling example of gene
editing’s potential for age
reversal2. The researchers
focused on a rare genetic
disorder called HutchinsonGilford Progeria Syndrome,
which causes rapid aging in
children. The disorder is usually
caused by a tiny change in the
DNA sequence of a speci昀椀c
gene. The team used a geneediting tool to correct this
change in skin cells. While the
correction was successful in only
about 25% of the cells, it was
suf昀椀cient to initiate a remarkable
process. The edited, healthy
cells began to multiply and
gradually took over, replacing
the cells that still had the genetic
SOCRA SOURCE © May 2024
anomaly. This demonstrates
how gene editing can set off a
chain reaction leading to overall
improvement, offering the
potential for reversing certain
age-related conditions.
Challenges & Solutions
in Genome Editing Trials:
Navigating the Path to
Age Reversal
The potential of genome
editing for reversing the aging
process is immense, but it’s not
without its challenges. One of
the biggest hurdles is ensuring
safety. Genome editing involves
making changes to our DNA,
which can have unintended
consequences and potentially
cause harm if not done
correctly4. The complexity
of the aging process itself
presents another challenge.
Aging involves many different
genes and biological pathways,
so it’s not as simple as just
changing one piece of our
DNA. Furthermore, there’s the
logistical challenge of delivery—
getting the genome editing
tools to the right cells in the
body is a major task.
Addressing the challenges
of genome editing requires
a multifaceted approach. To
mitigate off-target effects and
improve ef昀椀ciency, scientists
are continually re昀椀ning the
genome editing system,
developing more precise and
reliable versions. Advanced
computational tools are
also being used to predict
potential off-target sites and
optimize the design of the
guide RNAs, which direct the
genome editing system to the
correct location in the DNA.