Human Gene Editing And The CRISPR Revolution

A major medical milestone took place in May 2025, when
doctors at the Children’s Hospital of Philadelphia used
CRISPR-based gene editing to treat
a child with a rare genetic disorder. Unlike earlier CRISPR
(Clustered Regularly Interspaced Short Palindromic Repeats)
treatments that targeted well-known genetic mutations, this
marked a new level of personalized
medicine tailored to a patient’s unique DNA. For
advocates of biomedical innovation for
human enhancement, it was another sign of gene
editing’s vast potential, even as ethical, political, and
safety concerns remain.

Efforts to alter human genes
really began
in the 1970s, when scientists first learned to cut a
piece of DNA from one organism and attach it to another. The
process
was slow, imprecise, and expensive. Later
tools like meganucleases, transcription activator-like
effector nucleases, and zinc-finger nucleases improved
accuracy but remained technically complex and
time-consuming.

The real revolution came in
2012, when researchers Jennifer Doudna and Emmanuelle
Charpentier harnessed CRISPR, a natural bacterial defense
system. In bacteria, CRISPR cuts out invading viruses’ DNA
and inserts fragments into its own genome, allowing it to
recognize and defend against future infections. Doudna and
Charpentier showed that this process could be adapted to any
DNA, including human, creating a precise and programmable
system to target genetic mutations. Together with a protein
called CRISPR-associated protein (Cas9), which acts like
molecular scissors, it made cutting, modifying, and
replacing DNA faster, easier, and cheaper.

Attempts
to push the technology forward clashed with regulatory
caution and ethical debate, but more
than 200 people had undergone experimental CRISPR
therapies, according to a 2023 MIT Technology Review
article. The first major legal breakthrough came that
November, when the UK
approved Vertex Pharmaceuticals’ CASGEVY for the
treatment of transfusion-dependent beta thalassemia and
sickle cell disease. Enabled by advances in CRISPR
technology, CASGEVY works by making “an edit (or
‘cut’)… in a particular gene to reactivate the
production of fetal hemoglobin, which dilutes the faulty red
blood cells caused by sickle cell disease,” explained
Yale Medicine. Bahrain
and the
U.S. granted regulatory approval weeks later, and by
mid-2025, the EU and several other countries
followed.

CRISPR technology continues to advance, with
researchers at the University of Texas at Austin recently
unveiling a CRISPR therapy that can replace
large defective DNA segments and fix multiple mutations
simultaneously, overcoming the limits of traditional
one-site editing. “Epigenetic
editing,” meanwhile, uses modified Cas9 proteins to
turn genes on or off without cutting the DNA, and new CRISPR
systems can even insert
entirely new DNA directly into cells, bypassing the
cell’s natural repair process for larger precision
edits.

Alongside academic researchers, major companies
are emerging in the gene-editing field. By early 2025, the
U.S. had
217 gene-editing companies, compared with a few dozen in
Europe (mainly in the UK and Germany) and 30 in China,
according to the startup company BiopharmaIQ.

CRISPR
Therapeutics, Intellia Therapeutics, and Beam Therapeutics
are
among the industry’s leaders. A growing network of
companies and research teams attended the Third
International Summit on Human Genome Editing held in
London in 2023, following the first in Washington, D.C.,
in 2015, and the second in Hong Kong in 2018.

Smaller
companies are also innovating.
Xenotransplantation—transplanting nonhuman organs to
humans—has a long history, but CRISPR technology is
giving it new momentum. In 2024, Massachusetts General
Hospital transplanted a pig kidney edited with CRISPR-Cas9
technology to remove harmful pig genes and add human ones.
The pig kidney was provided by the American pharmaceutical
company eGenesis.

The
patient survived for two months before dying of unrelated
causes, and
the company completed another transplant in 2025. Other
companies, including United Therapeutics through its
subsidiary Revivicor, have begun their own trials in a
potential bid to transform the organ donor
industry.

CRISPR’s rapid spread has also fueled a
DIY biotech movement among transhumanists
and biohackers
interested in using biotechnology for human enhancement. Nonconventional
genetic experimentation, or “garage
research,” often outside standard regulation, has
become common. CRISPR kits can be ordered online for less than
$100, and their small size, relative
simplicity, and open-source
nature make experimentation and collaboration
possible.

“[N]ew technologies such as CRISPR/Cas9
give nonconventional experimenters more extensive gene
editing abilities and are raising questions about whether
the current largely laissez-faire governance approach is
adequate,” pointed
out a 2023 article in the Journal of Law and the
Biosciences.

One of the best-known figures in this
movement is former NASA biochemist Josiah Zayner, who
founded The ODIN in
2013 to sell CRISPR
kits “to help humans genetically modify themselves.”
Early efforts to showcase the scope and potential of this
technology proved
popular online, and in 2017, Zayner livestreamed
injecting CRISPR-edited DNA to knock out his myostatin gene
to promote muscle growth.

CRISPR has quickly expanded
beyond human experimentation. Mississippi dog breeder David
Ishee attempted to get regulatory approval for CRISPR
technology to prevent Dalmatians’ tendency to develop
bladder stones in
2017, but faced immediate regulatory pushback. The
agriculture sector has seen more luck: U.S. startup Pairwise
has developed
a CRISPR-edited salad mix for American consumers, and in
2024, a multinational biotech consortium began pilot
trials of drought-resistant maize in Africa.

China has
been a leading force in CRISPR innovation since its
inception. In 2014, Chinese researchers were
among the first to use CRISPR-Cas9 in monkey embryos,
and became the first to edit human embryos in
2015, drawing concern from international observers. In
2018, Chinese researcher He Jiankui altered
the DNA of two human embryos to make them immune to HIV.
Although the babies were
born healthy, the announcement caused international
outcry, leading to He’s three-year prison sentence in 2019
and stricter
Chinese regulations on human gene editing.

Chinese
companies and institutions are actively
pursuing international collaboration to solidify their
position. In August 2025, ClonOrgan
was part of a pig-to-human organ transplant, while other
Chinese entities established an early lead in CRISPR-based
cancer
therapies.

The U.S. and China remain clear leaders
in CRISPR research, and certain European countries are also
active, but others are also rapidly building capacity. In
April 2025, Brazil began the first
patient trial of CRISPR gene editing for inherited heart
disease, while growth has also been strong in Russia,
India,
and the Gulf
States.

Concerns and Inevitability

The
rapid adoption of CRISPR technology by private companies,
institutions, ideologists, and hobbyists globally has drawn
scrutiny. Despite the relatively low cost of developing
CRISPR therapies, the actual treatments remain expensive.
Social concerns have grown over the idea of “designer
babies,” where wealthier families could immunize their
children against diseases or select genetic traits,
exacerbating inequality.

The He Jiankui case, for
example, involved deleting the CCR5 gene in embryos to
prevent HIV, but may
have also improved their intelligence and memory due to
the link between CCR5 and cognition.

Safety concerns
also abound. Unintended downstream mutations, or “off-target
effects,” can cause genetic defects or chromosomal
damage, and in 2024, Swiss scientists documented
such issues, highlighting the risks of heritable
changes. Even DNA sequences once thought nonessential
may have important functions, and edits could have
unforeseen consequences for human evolution.

In 2015,
a group of leading scientists and researchers proposed
a global moratorium on heritable genome edits, yet
research has pressed on. Sterilized, genetically modified
mosquitoes were released in Africa to test population
control in
2019, and in 2020, Imperial College London demonstrated
that a “modification that creates more male offspring was
able to eliminate populations of malaria mosquitoes in lab
experiments.”

As with all emerging technologies,
CRISPR-based therapies are resulting in major legal
disputes. The Broad Institute, for example, holds patents
for using CRISPR in human and animal cells, while UC
Berkeley owns the original test-tube version, resulting
in a patent battle settled in 2022. “The tribunal of
the U.S. Patent and Trademark Office (USPTO) ruled that the
rights for CRISPR-Cas9 gene-editing in human and plant cells
belong to the Broad Institute of MIT and Harvard, not to
Berkeley,” stated an article
on the Cal Alumni Association website.

Biosecurity and
weaponization concerns also constrain greater CRISPR
adoption. Former U.S. Director of National Intelligence
James Clapper repeatedly
warned
that genome editing, including CRISPR, could be used as
weapons of mass destruction. Its ease of use has continued
to raise fears of manipulating pathogens or making
populations resistant to vaccines and treatments, as well as
the potential to enhance cognitive or physical abilities in
soldiers.

Still, the technology’s promise is too
significant to be overlooked, as reflected by the attention
it has received from Trump administration officials. Vice
President J.D. Vance spoke
positively about the CRISPR sickle cell treatment
shortly after being elected. Other administration figures
have financial ties to the industry, with disclosures showing
Robert F. Kennedy Jr.’s plans to divest holdings in
CRISPR Therapeutics AG and Dragonfly Therapeutics to avoid
conflicts of interest before taking office.

New CRISPR
tools, like base
editing and prime
editing, highlight the technology’s ongoing potential,
and in
2025, Stanford researchers and collaborators linked
these tools with AI to further augment their capabilities.
While consolidation among companies and institutions grows,
open-source labs may help drive a new frontier of innovation
that heavily regulated business and bureaucratic
organizations struggle to achieve.

CRISPR co-inventor
Jennifer Doudna wrote
in her 2017 book A Crack in Creation, “Someday
we may consider it unethical not to use germline
editing to alleviate human suffering.” With the potential
to cure
more diseases, some argue there is a moral obligation to
reduce avoidable suffering even amid ethical objections.
While companies have enormous financial incentives to bring
these therapies to market, government oversight, private
competition, and the eventual expiration of CRISPR patents,
which allow for wider access and lead to lower costs, will
be needed to ensure benefits
are widely shared as they unfold.

By John P.
Ruehl

Author Bio: John P. Ruehl is an
Australian-American journalist living in Washington, D.C.,
and a world affairs correspondent for the Independent Media
Institute. He is a contributor to several foreign affairs
publications, and his book, Budget Superpower: How Russia
Challenges the West With an Economy Smaller Than Texas’,
was published in December 2022.

This article was
produced by Economy for All, a project of the Independent
Media
Institute.

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