Human Gene Editing And The CRISPR Revolution – Analysis

November 14, 2025 
By John P. Ruehl


CRISPR-based technology is advancing rapidly, driving international competition. Its promise to transform medicine is colliding with political and social realities, even as applications expand.

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 approvedVertex 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 DNAdirectly 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 treatmentshortly 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 sharedas they unfold.

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.

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

REENGINEERING LIFE

Fresh Off Her Nobel Prize Win, Jennifer Doudna Predicts What’s Next for CRISPR

The new Nobel laureate chats with ‘Future Human’ about what her gene-editing companies are up to.


Emily Mullin

Photo illustration; Image source: picture alliance/Getty Images

Reengineering Life is a series from Future Human about the astonishing ways genetic technology is changing humanity and the world around us.

When the Royal Swedish Academy of Sciences announced on October 7 that she had won the 2020 Nobel Prize in chemistry, Jennifer Doudna was still fast asleep at home in California. It was just before 3 a.m. when a phone call woke her up. It was a reporter from Nature, asking if she could comment on the award.

“Well, who won it?” Doudna asked.

Doudna, PhD, of the University of California, Berkeley, and Emmanuelle Charpentier, PhD, of the Max Planck Institute in Germany, share the award for the discovery of the gene-editing technology CRISPR. The two biochemists began collaborating in 2011 and just a year later published a groundbreaking paper on CRISPR, which has revolutionized our ability to edit genes.

Short for clustered regularly interspaced short palindromic repeats, CRISPR is actually a naturally occurring bacterial immune system. When viruses attack bacteria, bacteria in turn grab snippets of genetic material from their viral invaders and incorporate these bits into their own DNA. This helps bacteria recognize viruses later on and thwart future invaders. Bacteria do this by producing an RNA molecule that acts as a guide, which cuts up the viral genome.

Doudna and Charpentier realized they could harness this cutting ability to edit genes in just about any living thing. In their 2012 paper, they described how this bacterial system could be used as “DNA scissors,” and the gene-editing technology CRISPR was born.

Declared as one of the most important discoveries of the 21st century, CRISPR is faster, cheaper, and more accurate than previous gene-editing systems and has since become ubiquitous in labs around the world. Scientists are using it in an attempt to treat serious genetic diseases, restore eyesight in people with a type of inherited blindness, engineer crops that are more resilient to disease and climate change, and eliminate disease-carrying pests like mosquitoes and mice. And researchers are already working on newer and improved versions of CRISPR that are even more precise.

The power to edit genes also opens up many ways for CRISPR to be abused. In 2018, Chinese scientist He Jiankui was widely condemned after revealing that he used CRISPR to make the world’s first known gene-edited babies. He is now serving a prison sentence, but the revelation has raised fears that CRISPR could lead to genetically enhanced “designer babies.”

After the Nobel announcement last week, I talked with Doudna about what’s next for CRISPR, the field of gene editing, and her own scientific work.

This interview has been lightly edited for grammar and clarity.

Future Human: First off, congratulations. What an incredible honor. How surprised were you to find out that you had won a Nobel prize?

Jennifer Doudna: Oh, total shock! I mean really. Coming out of a deep sleep and getting news like that — I couldn’t believe it. I said to the reporter who had called me, “I can’t talk to you right now. I have to call somebody and find out if this is official.”

We’re seeing a handful of clinical trials for CRISPR-based treatments get underway right now. What diseases do you see CRISPR being most promising for in the near future?

Certainly, diseases that are caused by single genes or genetic mutations. A great example, and we’ve already seen early results from one trial, is for sickle cell disease. But I think going forward, we’ll see opportunities to use CRISPR for other kinds of blood disorders, genetic diseases of the eye, and then, maybe in the longer term, cystic fibrosis and muscular dystrophy, which are also genetic diseases.

What do you think is going to be the biggest obstacle to getting these treatments to patients?

It’s probably delivery. One of the reasons why blood disorders have been some of the early targets of CRISPR is that the genome editing that’s used to correct those mutations can be done in cells that are taken out of a patient. The editing is done in the laboratory before reintroducing them versus a disease like cystic fibrosis or muscular dystrophy, where the editing would actually need to be done inside the body, in the right cells, to have a clinical benefit. That’s a hard challenge right now.

Your company, Mammoth Biosciences, is working on a rapid CRISPR-based test for Covid-19. What role do you think CRISPR diagnostic tests will play in the future?

There are several efforts underway to develop CRISPR diagnostics in comparable companies and academic labs. I think we’re going to see everything from high-throughput laboratory tests that require robotic equipment and experts to point-of-care tests that can be run in a research lab, a doctor’s office, or an emergency room. Down the road, we hope to have an at-home test that would work like a pregnancy test for Covid. What’s exciting with the CRISPR technology is that it’s potentially a faster and more direct way to detect the presence of the virus and also relies on a different supply chain than what’s necessary for the PCR (polymerase chain reaction) test.

What’s the status of your company’s Covid-19 test?

Mammoth Biosciences is planning on rolling out its test to a few partner labs for initial beta testing in November. Depending on how those experiments go and how those results turn out, we’ll expand to other labs after that. We want to see how it compares to the PCR test.

You recently just launched another CRISPR company, Scribe Therapeutics. What’s the focus of this new startup?

This is the thing about CRISPR: There’s so many different ways that it can be deployed. For clinical applications, the reason we’re seeing a lot of early efforts focused on blood disorders like sickle cell disease and, to some extent, diseases of the eye or even the liver is because those tissues are easier to introduce gene-editing molecules into. With Scribe Therapeutics, we’re looking at opportunities to use CRISPR for neurodegenerative diseases. For those disorders, the technology obviously needs to be very robust and very safe. It also has to get into brain cells and neural tissue where it can have an impact. We want to make sure that the editing tools are the best they can be and then figure out the best way to introduce them into the brain. That’s really the focus of the company.

What do you think is going to be the next big CRISPR advance?

That’s always a hard question. We’ve so much going on in the field. I think one interesting possibility is that we’ll see CRISPR being used not to edit genomes, or at least not to make permanent changes to genomes, but instead to regulate them, to control levels of human proteins that are produced from different genes. This is a newer way of using the CRISPR technology. I think it has a lot of potential to allow control of cells that doesn’t require actual permanent chemical changes being made to the DNA.

After the birth of the CRISPR babies in China in 2018, there’s been a lot of talk around the idea of germline or heritable genome editing. Do you think that should be off-limits to scientists right now?

I don’t think it needs to be completely off-limits. I was pretty pleased with the recent report that came out from the National Academies and the U.K. Royal Society that recommends a kind of a measured approach to developing the technology for use in the human germline. They’re encouraging research to understand how the technology works in embryos. First, the technology will need to be proven safe. Secondly, any clinical use [to establish a pregnancy] would need to be restricted to cases of serious genetic disease where there are few or no other options to treat the disease. I think those are both pretty high bars. Those situations are pretty rare. I personally think there are more viable strategies today, like embryo screening and selection in an IVF (in vitro fertilization) clinic, rather than using genome editing.

The report you mentioned also calls for “extensive societal dialogue” before countries decide to permit the use of heritable human genome editing. You’ve talked in the past about public engagement around CRISPR. How do we educate and engage the public about CRISPR?

Yeah, that’s really critical. I think the media has an important role to play in terms of large-scale education. Interactive media, like videos and documentaries, can also help. The challenge of course is making sure that the science is right.

How do we make sure that the public’s voice is heard in regard to how CRISPR is used?

It’s a really important question. It’s a challenge because, on one hand, I think it’s critical to have more public engagement in important decisions like this about how technology is used. On the other hand, that requires a level of understanding about the technology that the average person might not have or maybe doesn’t want to have. So, I think it’s important to have different formats and forums for encouraging discussion. We’ve already seen this with CRISPR in some way. On the one hand, there are highly technical meetings that include discussion of ethical and societal issues from a pretty detailed technical standpoint. But there are also increasingly conferences and events that don’t get into the weeds of the science per se, but they spend a lot more effort thinking through CRISPR’s implications and its different uses. Inviting people who are nonspecialists to engage in those meetings has been really effective.

Other than medicine, where else do you think CRISPR could be transformative?

Agriculture is the other area where it’s going to be impactful. We’re already seeing a lot of use of CRISPR in making plants that have genetic changes that can enable things like better crop yield, resistance to drought, higher levels of nutritional value, things like that. I think that’s really exciting, and there’s clearly a lot more to be done there. That’s likely to be the area where we’ll see a broader impact in the near term.

Given CRISPR’s potential for misuse, how do you think it should be regulated?

Fortunately, I think there’s quite a good regulatory framework in the United States and in most places that have major research operations that can serve to regulate the use of CRISPR. That really goes back to the 1970s [at the Asilomar Conference on Recombinant DNA], when voluntary guidelines were put in place for using some of these early tools of molecular biology, like molecular cloning. That being said, as we discussed, there are certain applications of CRISPR like in human embryos, where I think there needs to be special attention.

Who are the CRISPR scientists you really admire?

Many. It’s become a huge field. It used to be tiny, and now it’s vast. One is Luciano Marraffini. He’s a scientist at Rockefeller University. He works on the fundamentals of CRISPR biology and understanding how it works in bacteria as an immune system. Jill Banfield at Berkeley is continuing to do work on bacteria that are not cultured in the lab but are growing in various environmental niches. She was one of the very early discoverers of CRISPR systems and bacteria, and she continues to find a lot of new ones. In plant biology, I really like the work of Pamela Ronald at the University of California, Davis. She’s doing work primarily in rice, where they are using CRISPR to make modifications to the rice genome that I think are going to be really important as rice farmers face the challenges of climate change. On the biomedical side, I’m really excited about the work of Charles Gersbach at Duke University.

What’s next for you?

CRISPR is going to keep us busy for a while. There are still a lot of fundamental questions about how these pathways operate that I really like to try to answer. Jill Banfield is a close collaborator of ours, and she continues to supply us with many, many new CRISPR pathways that we’re excited to investigate. We’re also really interested in genome editing in natural microbial communities. I think there’s a really interesting opportunity to be able to manipulate certain microbes. I think those are areas where I’ll be focusing my efforts in the near term.


WRITTEN BY
Emily Mullin
Staff writer at OneZero and Future Human, where I cover biotechnology and genetic privacy. I also teach science writing at Johns Hopkins.

Future Human is a science publication from Medium about the survival of our species. It is run by the OneZero editorial team, which also publishes stories about major forces in technology through its namesake publication and stories about gadgets on Debugger.