From the potential capabilities of manipulating
DNA to the irresponsible mistakes that can
occur, today we look at The Truth About CRISPR
& Gene Editing.
Number 9.
CRISPR Unraveled
Associated recently with the concept of gene
editing, the term CRISPR actually stands for
Clustered Regularly Interspaced Short Palindromic
Repeats.
What this refers to specifically are the DNA
sequences found in bacteria that act as an
immune system.
This DNA holds onto the bits of invading viruses
the bacteria has already eradicated.
The Cas9 enzymes produced by the bacteria
to fight viruses will use these stored bits
to identify past infiltrators and dispose
of them quickly.
For years, this information was simply a scientific
discovery useful to microbiologists, but what
researchers found later quickly changed that
forever.
Number 8.
Revolutionary Advancement
In 2011, two researchers were enamored by
the CRISPR process and the Cas9 enzymes and
began examining them in detail.
The University of California Berkeley's Jennifer
Doudna and Umea University's Emmanuelle Charpentier
wanted to know how, exactly, the Cas9 enzymes
chose what viruses to chop up and store and
when to do so.
In their experiments, they found they could
actually fool the bacteria's CRISPR and Cas9
enzymes and give it artificial RNA.
From here, the Cas9 proteins would store that
information upon shredding the RNA and use
the stored data to attack anything with the
same genetic code.
So a year later, the team of scientists released
a paper detailing their work and the conclusion
that they could, in a way, cut up any genome
anywhere they want.
The discovery was mind blowing to the genetic
research community.
It wasn't long until inspired scientists,
like Feng Zhang [fung jong] of the Broad Institute
and George Church of Harvard, would discover
new uses for this discovery...like the editing
of human cells.
Not only would they be able to remove harmful
genes with the Cas9 protein...but replace
them with beneficial ones as well.
And it does so at a low cost and incredible
precision, advancing gene editing in multiple
ways.
Number 7.
Beneficial Uses
The discovery of what CRISPR can control and
do has sent minds racing and some of the potential
advancements that could come from using their
enzymes are world-changing.
For example, scientists have had a map of
the human genome since 2003, but understanding
and identifying what each gene does continues
to elude geneticists.
But CRISPR will allow scientists to figure
out what each gene does by systematically
removing them and noting the resulting effect.
That's only the beginning, though.
Using CRISPR , scientists could complete such
feats as creating more nutritious and even
tastier crops.
Farmers could herd hornless dairy cows.
Peanuts could be harvested sans allergens.
Even fruits themselves could have their immunity
strengthened to survive fungal diseases or
other afflictions.
CRISPR could also be used to take out genetic
diseases and even be used to remove HIV infections.
It could also be used to exterminate bacteria
more effectively as they build stronger and
stronger resistances to antibiotics.
Animals that carry potentially dangerous diseases
or they themselves prove to be too lethal
could be altered for the better with CRISPR
. And then there's babies.
While they can have their DNA edited by CRISPR
to be resistant to diseases and viruses, the
other side of this coin holds implications
far more sinister.
Number 6.
Ethical Dilemmas
While the benefits of being able to eradicate
genes that might cause harm to a baby later
in life sound grand, there are negative implications
that come with the power to edit your newborn's
DNA.
Scientists are far off from being able to
recognize and alter genes that would, for
instance, reliably raise your child's intelligence.
They have yet to identify even the most dire
of threats to children in terms of their gene
pool, let alone enhance specific characteristics.
As far as the science community at large is
concerned, there is not enough known about
using CRISPR on the human genome to guarantee
a safe result or even a beneficial one.
But this doesn't mean attempts can't be made.
CRISPR technology is relatively inexpensive
and because of that, experiments can be conducted
fairly easily, no matter how rebellious to
scientific wisdom they may be.
In the past, genetically modified organisms,
or GMOs, have raised public suspicion due
to a lack of transparency thus creating public
distrust.
The geneticists behind the advancement of
CRISPRs have hoped to avoid the same issue,
but irresponsible procedures, like those performed
by one Chinese researcher in particular, put
those hopes in jeopardy.
Number 5.
Unnecessary Experiment
In November of 2018, the global scientific
community was rattled with the news that He
Jiankui , a young genetic researcher from
China, had performed the first CRISPR -edit
on babies, a pair of twin girls named Nana
and Lulu.
He described his experiment in person at the
Second International Summit on Human Genome
Editing held at the University of Hong Kong
and immediately drew the ire of fellow scientists,
including the CRISPR trailblazer Jennifer
Doudna . In her response, Doudna said, "I
feel deeply disappointed that the technology
was used in the way that he described as well
as a bit horrified, honestly."
Firstly, the aim of He's experiment was misguided.
He and his team intended to remove a gene
used by HIV viruses to enter human cells once
the HIV virus was contracted.
The only problem was that while their father
may have been HIV-positive, the twins didn't
have the virus.
And even if they did, there were already safer
proven treatments that could've done the same
thing.
To make matters worse, He allegedly didn't
edit the genes correctly or even completely.
Instead, new mutations seem to have appeared
in their genetic code!
Even the details of his experiment, which
were only revealed to a select few at the
Hong Kong summit, were said to have been amateurish
by other scientists.
Number 4.
Unintended Consequences
While he may have had good intentions, the
results of He's [h'uz] work are far from successful.
Two of the three mutations introduced to Nana
and Lulu changed their CCR5 gene, the one
targeted in association with HIV, significantly.
The results of such mutations could alter
how they work and the outcome of their mutation
is something that will need to be studied.
Normally, this kind of experiment should have
been performed on lab animals before moving
on to human subjects, at which point consenting
adults afflicted with HIV could sign up for
the experimental procedure.
But He [h'u] catapulted over these logical
steps, instead implanting the CRISPR -edited
genes into the embryos of a woman.
This wasn't the only ethically questionable
part of He's [h'uz] experiment, though.
In order to find a patient for this project,
He [h'u] utilized an AIDS association to find
potential subjects under the guise of an "AIDS-vaccine
development project."
The scientist claims to have gotten the consent
of the individuals in question, however taking
consent is a skill that He [h'u] was unqualified
to perform, thus calling the validity of their
consent into question.
The consent form used for his patients is
even problematic as the wording is overly
technical and unclear of any dangers or controversy
surrounding the project.
Instead, it focuses more on securing the protection
of He [h'u] and his team from any legal blowback
as well as the right to use photos of the
patients for advertising purposes.
Number 3.
Saving Face
As if predicting the backlash he'd receive
for such a project, He [h'u] took certain
steps prior to conceal his work.
His own school, the Southern University of
Science and Technology, were never informed
of his ambitions and in order to keep his
work unassociated with the college, he took
unpaid time off to begin his pursuit.
The renegade researcher does claim, however,
to have received ethical approval from the
Shenzhen Harmonicare Hospital.
The hospital's Medical Ethics Committee, however,
disputes this statement, going on to clarify
that the signature's on He's [h'uz] supposed
approval form "are suspected to have been
forged".
In hopes of combating the condemnation received
by contemporaries, He [h'u] organized a PR
campaign to clear his name.
Hiring American PR consultant Ryan Ferrell,
He [h'u] crafted a series of five videos for
YouTube in which the scientists elaborates
on his motivations and logic behind the experiment,
essentially trying to give the public his
side of things.
The videos themselves have gained some traction,
with one of the videos, specifically the clip
made concerning the twins Lulu and Nana, achieving
more than 400 thousand views online.
Though a mixed reception in the comments section
reveals He Jiankui still has many more minds
to sway.
Number 2.
On The Take
The news of He Jiankui's experiment shocked
most everyone in the science community, though
there were some with knowledge of the rogue
scientist's intent before his unveiling to
the world.
He [h'u] had made his field of study known
through various scientific conferences where
he had shared gene-editing research in other
animals.
But secretly, he would share his true endeavors
with only a select few scientists.
This included his former adviser, Rice University's
Michael Deem, who participated actively with
the gene-editing product, going so far as
to have been present during patient consenting.
That would be the extent of support that He
could garner, though, as most other scientists
and researchers weren't nearly as encouraging.
When attempting to seek advice from experts
like Matthew Porteus and Stephen Quake of
Stanford and Mark DeWitt of UC Berkeley, the
response was unanimously disapproving.
Chided for his naivete in regards to the ethical
implications, He was told by all three not
to go through with the project.
To counteract such blatant acts of ethical
defiance in the science community, David Baltimore,
chair of the Hong Kong summit on genome editing,
has called for a "see something, say something"
policy among scientists.
While no single international group oversees
the ethical use of gene-editing, countries
do have their own means to regulate the procedures,
like China's medical-ethics agency.
Number 1.
Scientific Aftermath
Since news of He's [h'uz] experiment hit the
public, various scientists, including stem-cell
biologist Paul Knoepfler and Feng Zhang [fung
jong] of the Broad Institute, have called
for similar experiments to be temporarily
prohibited.
Originally, the Hong Kong summit released
a statement with a fairly lax connotation
but a second statement came out following
the summit's conclusion, calling He Jiankui's
supposed experiment "irresponsible" and the
results "deeply disturbing".
However, the summit and similar-minded scientists
still believe genome editing is the future,
with the summit going on to say: "The organizing
committee concludes that the scientific understanding
and technical requirements for clinical practice
remain too uncertain and the risks too great
to permit clinical trials of germline editing
at this time.
Progress over the last three years and the
discussions at the current summit, however,
suggest that it is time to define a rigorous,
responsible translational pathway toward such
trials."
