CRISPR and Cancer: How Gene Editing Is Revolutionizing Oncology
Here's the problem with that fantasy. Cancer isn't a clean typo. It's a riot. It's a chaotic, messy, ever-changing system that thrives on mistakes. It rewrites its own rulebook as it goes. So you have this exquisitely precise scalpel, and you're trying to use it in the middle of a cellular brawl [7]. It's a mismatch. A big one.
This isn't me being cynical. It's just recognizing where the real work is. And right now, the most revolutionary thing CRISPR is doing for cancer has nothing to do with curing patients directly. It's happening in the lab [1]. For years, if we wanted to know what a specific gene did, we had to go through a long, painful process to shut it down. Now? A researcher can just use CRISPR to snip that gene out of a cancer cell in a petri dish and see what happens [4]. Does the cell die? Stop growing? Suddenly become vulnerable to an old drug? We can get answers in weeks, not years. We're building a proper instruction manual for our enemy, and we're doing it at a shocking speed [5]. That's the real revolution so far. It's a research tool. And it's the best one we've ever had.
So if we can't just march into the body and 'edit' the tumor away, what's the next best thing? You take the fight somewhere else. Somewhere you can control. That's the whole idea behind CAR-T cell therapy. We take a patient's own immune cells—their T-cells—out of their body and into the lab. We engineer them to recognize cancer. Then we put them back. It can work miracles. It can also fizzle out. The T-cells get tired. The cancer learns to hide.
This is where CRISPR gets its first real clinical shot. In the lab, before we put those T-cells back, we can give them a CRISPR tune-up. A few quick snips. We can edit out the gene that tells a T-cell to stand down, making it relentless at it's goal. We can snip out its natural receptors so it doesn't get confused and attack the wrong thing [3]. Better weapons. Stronger armor. A clearer target [10]. We're not fixing the patient. We're upgrading their soldiers in the workshop before sending them to the front lines. This is where the action is right now, especially for nasty, hard-to-treat cancers, like in gynecological oncology or tumors that have stopped responding to anything else [6] [7]. It's practical. It's contained. And it's already happening.
But the sci-fi dream of injecting a cure dies hard. Why can't we just do that? It comes down to boring, practical problems. The kinds of problems that kill big ideas. Delivery and safety [3] [9]. First, how do you get the CRISPR machinery into every last cancer cell, deep inside a person's body, without also hitting a bunch of healthy cells by mistake? Our delivery systems just aren't that good yet. It's like trying to mail a million letters and making sure every single one gets to the right house during an active hurricane.
Then there's the 'oops' factor. Off-target effects. What if the scissors cut the wrong gene [8]? In the best case, nothing happens. In the worst case, you create a whole new problem. You could, in theory, cause a new cancer while trying to cure the old one. The risk is small. It's getting smaller every year. But it's not zero. And when you're talking about rewriting someone's permanent code, the stakes are as high as they get.
So, no. This isn't a magic wand. The revolution is a slow, grinding one. It's happening at the bench, letting us understand the disease. It's happening in the cell processing lab, letting us build better therapies. The idea of a simple cure-in-a-needle remains a distant hope.
[1] Wang, S. W., Gao, C., Zheng, Y. M., Yi, L., Lu, J. C., Huang, X. Y., Cai, J. B., Zhang, P. F., Cui, Y. H., & Ke, A. W. (2022). Current applications and future perspective of CRISPR/Cas9 gene editing in cancer. Molecular cancer, 21(1), 57. https://doi.org/10.1186/s12943-022-01518-8
[2] Garg, P., Singhal, G., Pareek, S., Kulkarni, P., Horne, D., Nath, A., Salgia, R., & Singhal, S. S. (2025). Unveiling the potential of gene editing techniques in revolutionizing Cancer treatment: A comprehensive overview. Biochimica et biophysica acta. Reviews on cancer, 1880(1), 189233. https://doi.org/10.1016/j.bbcan.2024.189233
[3] Shuvalov, O., Petukhov, A., Daks, A., Fedorova, O., Ermakov, A., Melino, G., & Barlev, N. A. (2015). Current genome editing tools in gene therapy: new approaches to treat cancer. Current gene therapy, 15(5), 511–529. https://doi.org/10.2174/1566523215666150818110241
[4] Zhao, Z., Li, C., Tong, F., Deng, J., Huang, G., & Sang, Y. (2021). Review of applications of CRISPR-Cas9 gene-editing technology in cancer research. Biological procedures online, 23(1), 14. https://doi.org/10.1186/s12575-021-00151-x
[5] Chehelgerdi, M., Chehelgerdi, M., Khorramian-Ghahfarokhi, M., Shafieizadeh, M., Mahmoudi, E., Eskandari, F., Rashidi, M., Arshi, A., & Mokhtari-Farsani, A. (2024). Comprehensive review of CRISPR-based gene editing: mechanisms, challenges, and applications in cancer therapy. Molecular cancer, 23(1), 9. https://doi.org/10.1186/s12943-023-01925-5
[6] Kumar N. (2025). Genome Editing in Gynecological Oncology: The Emerging Role of CRISPR/Cas9 in Precision Cancer Therapy. Therapeutic innovation & regulatory science, 10.1007/s43441-025-00807-w. Advance online publication. https://doi.org/10.1007/s43441-025-00807-w
[7] Mishra, G., Srivastava, K., Rais, J., Dixit, M., Kumari Singh, V., & Chandra Mishra, L. (2024). CRISPR-Cas9: A Potent Gene-editing Tool for the Treatment of Cancer. Current molecular medicine, 24(2), 191–204. https://doi.org/10.2174/1566524023666230213094308
[8] Imyanitov, E. N., Iyevleva, A. G., & Levchenko, E. V. (2021). Molecular testing and targeted therapy for non-small cell lung cancer: Current status and perspectives. Critical reviews in oncology/hematology, 157, 103194. https://doi.org/10.1016/j.critrevonc.2020.103194
[9] Uddin, F., Rudin, C. M., & Sen, T. (2020). CRISPR Gene Therapy: Applications, Limitations, and Implications for the Future. Frontiers in oncology, 10, 1387. https://doi.org/10.3389/fonc.2020.01387
[10] Murty, T., & Mackall, C. L. (2021). Gene editing to enhance the efficacy of cancer cell therapies. Molecular therapy : the journal of the American Society of Gene Therapy, 29(11), 3153–3162. https://doi.org/10.1016/j.ymthe.2021.10.001
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Business Wire
5 hours ago
- Business Wire
Profluent Announces Publication of Generative AI Research in Nature with New Results for OpenCRISPR-1
EMERYVILLE, Calif.--(BUSINESS WIRE)--Profluent, the AI-first protein design company, today announced a publication in Nature describing the design of OpenCRISPR-1, the world's first AI-created genome editor to successfully edit the human genome. These findings validate the use of Profluent's large language models (LLMs) to generate custom gene editors that match or exceed the performance of naturally occurring CRISPR systems, with the potential to expand gene editing capabilities and accelerate therapeutic development. Despite the diversity of CRISPR-Cas proteins that exist in nature, not all are ideally suited for specific therapeutic applications, and traditional protein engineering approaches introduce tradeoffs that limit utility. The publication, titled 'Design of highly functional genome editors by modelling CRISPR–Cas sequences,' explores Profluent's approach to AI-enabled protein design as a compelling alternative to directed evolution and structure-guided mutagenesis for engineering novel gene editing systems at scale. 'As our Nature publication demonstrates with the highest scientific rigor, the novelty and diversity of the gene editors Profluent can create with our LLMs transcend that of proteins made by nature or traditional engineering,' said Ali Madani, Ph.D., Profluent co-founder and Chief Executive Officer. 'By opening up this new frontier in AI-powered drug discovery, we are shifting biology from a field constrained by evolution to one of abundant possibilities, where we can generate new proteins on demand to solve society's most pressing challenges.' The paper expands on a previously published preprint with several new findings that reinforce the effectiveness of AI for protein design: AI generates sequences with unprecedented novelty and diversity: LLMs trained on large-scale biological sequences produce diverse CRISPR-Cas sequences spanning a broad set of families, while being hundreds of mutations away from known natural proteins and therefore impossible to create manually. LLMs trained on large-scale biological sequences produce diverse CRISPR-Cas sequences spanning a broad set of families, while being hundreds of mutations away from known natural proteins and therefore impossible to create manually. Viability of LLMs for CRISPR-Cas design: Profluent's platform performed exceptionally well in designing functional Cas proteins compared to manual or other machine learning approaches to protein engineering tested. Profluent's platform performed exceptionally well in designing functional Cas proteins compared to manual or other machine learning approaches to protein engineering tested. Genome-wide off-target analysis of OpenCRISPR-1: OpenCRISPR-1 showed significantly reduced off-target cleavage events using an unbiased genome-wide specificity assay. OpenCRISPR-1 showed significantly reduced off-target cleavage events using an unbiased genome-wide specificity assay. Lower potential immunogenicity with OpenCRISPR-1: iELISA quantification showed lower immunogenicity for all generated Cas proteins compared to SpCas9, suggesting that new-to-nature proteins designed with machine learning have the potential to be less immunogenic as compared to pathogen-derived genome editors, like SpCas9. 'Since releasing OpenCRISPR-1 in 2024, tens of thousands of academic and industry researchers have accessed the open-source sequence, signaling AI's broad potential to unlock solutions across therapeutics, biomanufacturing, agriculture, climate, and beyond,' said Hilary Eaton, Ph.D., Profluent Chief Business Officer. 'As we deepen investment in our platform capabilities to meet the bespoke needs of our partners, we're building Profluent into a one-stop-shop for customized gene editors of any modality, with attractive economics and flexible partnering models.' OpenCRISPR-1 is publicly available and free to license for ethical research and commercial use. The open-source package can be accessed via GitHub and AddGene. Profluent has also decided to open-source the CRISPR-Cas Atlas, the most extensive dataset of CRISPR systems curated to date, that was used to steer Profluent's models. This move aims to further expand access to Profluent's next-generation gene editing capabilities. About Profluent Profluent is an AI-first company pushing the frontier of de novo protein design to author new biology. Grounded in nature with AI as an interpreter, Profluent's powerful foundation model platform unlocks solutions that transform medicine, agriculture, and beyond. Founded in 2022 and headquartered in Emeryville, CA, Profluent is backed by leading investors including Spark Capital, Insight Partners, Air Street Capital, AIX Ventures, and Convergent Ventures. To learn more, visit
CNN
6 hours ago
- CNN
Could stem cells be used to create life without sperm or egg? Not yet, but here's why scientists are concerned
Maternal health Women's healthFacebookTweetLink Follow Scientists are exploring ways to mimic the origins of human life without two fundamental components: sperm and egg. They are coaxing clusters of stem cells – programmable cells that can transform into many different specialized cell types – to form laboratory-grown structures that resemble human embryos. These embryo models are far from perfect replicas. But as labs compete to grow the best likeness, the structures are becoming increasingly complex, looking and behaving in some way as embryos would. The structures could further the study of human development and the causes infertility. However, the dizzying pace of the research, which started little more than a decade ago, is posing ethical, legal and regulatory challenges for the field of developmental biology. 'We could have never anticipated the science would have just progressed like this. It's incredible, it's been transformative how quickly the field has moved, said Amander Clark, a professor of molecular cell and developmental biology at the University of California, Los Angeles, and the founding director of the UCLA Center for Reproductive Science, Health and Education. 'However, as these models advance, it is crucial that they are studied in a framework that balances scientific progress with ethical, legal and social considerations.' Clark is co-chair of the International Society of Stem Cell Research (ISSCR) Embryo Models Working Group, which is now trying to update such a framework on a global scale. At issue is the question of how far researchers could go with these stem cells, given time and the right conditions. Could scientists eventually replicate an actual embryo that has a heartbeat and experiences pain, or one that could grow into a fully developed human model? As current research stands, no model mimics the development of a human embryo in its entirety — nor is any model suspected of having the potential to form a fetus, the next stage in human development equivalent to week 8 or day 56 in a human pregnancy. Creating embryo models has also been a hit-and-miss process for most research groups, with only a small percentage of stem cells going on to self-organize into embryo-like structures. However, the models do exhibit several internal features and cell types that an embryo needs to develop, such as the amnion, yolk sac and primitive streak, and that could, 'with future improvements, eventually progress toward later embryo structures including heart, brain, and other organ rudiments,' according to a June paper coauthored by Clark and published in the journal Stem Cell Reports. Similar models made with mouse cells have reached the point where the brain begins to develop and a heart forms. Nobel laureate Jennifer Doudna tells Fareed about her path to becoming a leading scientist and explains how her discovery of CRISPR can help cure diseases and improve crops. Critically, the goal isn't to develop these models into viable fetuses, ultimately capable of human sentience, but to develop a useful research tool that unlocks the mysteries of how a human cell divides and reproduces to become a human body. The models also make way for experiments that can't be performed on donated embryos in a lab. However, it's possible as research advances that the distinction between a lab-grown model and a living human embryo could become blurred. And because the models lie at the intersection of historically controversial fields — stem cell biology and embryology — the work merits closer oversight than other forms of scientific research, Clark said. Clark and the ISSCR's Embryo Models Working Group in June recommended enhanced oversight of research involving the models. The society's guidelines, which first included guidance on embryo models in 2021, are being revised to incorporate the recommendations of the group and will be released in a few weeks. The current ISSCR guidelines make a distinction between 'integrated embryo models' that replicate the entire embryo, and 'non-integrated models' that replicate just one part of an embryo, requiring stricter oversight of the former. The updated guidelines will instead recommend that all research involving both types of embryo models should undergo 'appropriate ethical and scientific review.' The proposed update will also set out two red lines: The current guidance already prohibits the transfer of human embryo models into a human or animal uterus. The updated version will also advise scientists not use human embryo models to pursue ectogenesis: the development of an embryo outside the human body via the use of artificial wombs — essentially creating life from scratch. According to Clark, the stem cell-based embryo models she and other research teams work on should be considered distinct from research on actual human embryos, usually surplus IVF embryos donated to science. Such research is tightly regulated in many countries, and banned in others, including Germany, Austria and Italy. It makes sense, at least for now, to treat models and real embryos differently, said Emma Cave, a professor of healthcare law at Durham University in the UK who works on embryo models. She uses diamonds as an analogy: Natural diamonds and their commercially lab-grown equivalents are made from the same chemical components, but society assigns them different values. She cautioned there shouldn't be a rush to regulate embryo models too quickly in case it shuts down promising research. 'We are at an early stage in their development, where it could be that in 5, 10, 15, 20 years, that they could look very like a human embryo, or it might be they never get to that stage,' she said. As the scientific research unfolds, oversight of embryo models is taking different shapes in different jurisdictions. Australia has taken the strictest approach. It includes embryo models within the regulatory framework that governs the use of human embryos, requiring a special permit for research. The Netherlands in 2023 similarly proposed treating 'non-conventional embryos' the same as human embryos in the eyes of the law. The proposal is still under discussion, according to the Health Council of the Netherlands. Researchers in the United Kingdom released a voluntary code of conduct in 2024, and Japan has also issued new guidelines governing research in the field. In the United States, embryo models aren't covered by any specific legal framework, and research proposals are considered by individual institutions and funding bodies, Clark noted. The National Institutes of Health said in 2021 that it would consider applications for public funding of research into embryo models on a case-by-case basis and monitor developments to understand the capabilities of these models. Few other countries, however, appear poised to adopt specific legislation on embryo models, making the guidelines issued by the ISSCR a 'highly influential' reference for researchers around the world, according to the Nuffield Council on Bioethics, a London-based organization that advises on ethical issues in biomedicine. The council said in a November 2024 report that international guidelines were key to avoid 'research being carried out that does not meet high ethical and scientific standards; this in turn could impact on the national public perception of risk, leading to a more risk-averse approach that hinders responsible scientific development.' Clark said the ISSCR's updated voluntary guidelines would help scientific funding bodies around the world better evaluate applications and publishers of research understand whether work was performed in an ethically responsible way, particularly in places where the law or other guidelines don't take embryo models into account. The future challenge for regulators is to understand when and whether an embryo model would be functionally the same as a human embryo and therefore potentially afforded the same or similar protection as those surrounding human embryos, said Naomi Moris, group leader at The Francis Crick Institute's developmental models laboratory. The only definitive test would be to transfer the model into the uterus of a surrogate, a move that's forbidden by current bioethical standards. However, Moris is among a group of researchers that has proposed to two tipping points or 'Turing tests' — inspired by computer scientist Alan Turing's way of determining whether machines can think like humans — to evaluate when distinctions between a lab-gown model and a human embryo would disappear. 'These things are not embryos at the moment, they clearly don't have the same capacity as an embryo does. But how would we know ahead of time that we were approaching that?' Moris said. 'That was the logic behind it. What metrics would we use as a kind of proxy for the potential of an embryo model that might then suggest that it was at least approaching the same sorts of equivalency as an embryo.' The first test would measure whether the models can be consistently produced and faithfully develop over a given period as normal embryos would. The second test would assess when animal stem cell embryo models — particularly animals closest to humans such as monkeys — show the potential to form living and fertile animals when transferred into surrogate animal wombs, thus suggesting that the same outcome would in theory be possible for human embryo models. That hasn't happened yet, but Chinese researchers in 2023 created embryo models from the stem cells of macaque monkeys that when implanted in a surrogate monkey triggered signs of early pregnancy. Proponents of the technology say the models offer an equally, and possibly more, useful, ethical alternative to research on scarce and precious human embryos. The models have the potential to be produced at scale in a lab to screen drugs for embryo toxicology, a impactful application given that pregnant women have often been excluded from drug trials because of safety concerns. Yet, the potential for these models to be used in the creation of life has been cause for worry among bioethicists. 'There are commercial and other groups raising the possibility of building an embryo in vitro and combining different bioengineering approaches to bring such an entity to viability,' according to the June paper coauthored by Clark and other members of the ISSCR's embryo model working group. 'Currently the practice of bringing an SCBEM (stem cell-based embryo model) to viability is considered unsafe and unethical and should not be pursued,' the study noted. Cave said ectogenesis may sound like the realm of science fiction, but it isn't impossible. As embryo models continue to be developed, and separate research is advancing into artificial wombs, the two technologies could meet, Cave said. The challenge, she added, is recognizing the value of these research paths but at the same time preventing misuse. Jun Wu, an associate professor at the Department of Molecular Biology at the University of Texas Southwestern is one of a number of stem cell biologists involved in the field. He agreed that ectogenesis should be off the table but explained that researchers developing embryo models must engage in a delicate dance: To the unlock the mysteries of the human embryo, models have to resemble embryos closely enough to offer real insight but they must not resemble them so closely that they risk being viewed as viable. Magdalena Zernicka-Geotz, the Bren professor of biology and biological engineering at Caltech, said she welcomed the new guidelines. She announced in 2023 that her team had succeeded in a world first: growing embryo-like models to a stage resembling 14-day-old embryos. Later the same year, Jacob Hanna, a professor of stem cell biology and embryology at the Weizmann Institute of Science in Israel, said his team had gone a step further with a model derived from skin cells that showed all the cell types that are essential for an embryo's development — including the precursor of the placenta. Together the work represented a breakthrough for the models' potential use in research on pregnancy loss: At 14 days the human embryo has begun to attach to the lining of the uterus, a process known as implantation. Many miscarriages occur around this stage, Zernicka-Geotz said. Lab research on human embryos beyond 14 days, including those donated from IVF treatments, is prohibited in most jurisdictions. And while some scientists do study tissue obtained from abortions, such tissue is limited because few procedures take place between week 2 and week 4 of an embryo's development. The ability to grow an embryo model outside of a womb at this developmental stage paves the way for studies that are not possible in living human embryos. 'Far more pregnancies fail than succeed during the critical window just before, during and immediately after implantation. This is why we created in my lab the embryo-like structures from stem cells as a way to really understand this critical and so highly fragile stage of development,' Zernicka-Goetz said. Clark agreed that embryo models could potentially be used to address infertility problems: 'Implantation. It's the big black box. Once the embryo implants in the uterus, we understand very little about the development,' Clark added. 'And if we can't study it, we don't know what we're missing.'
Business Wire
9 hours ago
- Business Wire
Aldevron Launches New Innovation Center in Waltham, MA
BUSINESS WIRE)--Aldevron, a Danaher company, and a global leader in the production of DNA, RNA and protein in the genomic medicine space, today announced the planned opening of its new Innovation Center in Waltham, Massachusetts. The milestone marks a bold step in Aldevron's mission to accelerate innovation in genomic medicines while deepening its presence in one of the world's most dynamic biotech hubs. "Establishing a presence in Waltham puts Aldevron at the crossroads of scientific discovery.' - Venkta Indurthi, CSO of Aldevron Share The new Waltham site is designed to fast-track advancements in the manufacture of DNA, RNA and proteins for advanced therapies, expanding Aldevron's capabilities in cell-free DNA, molecular biology, gene editing, and mRNA analytics. By establishing a base in the Boston area, Aldevron is positioning itself at the epicenter of biotech innovation—enabling closer collaboration with clients, faster project timelines and enhanced support for the development of advanced therapies. 'Establishing a presence in Waltham puts Aldevron at the crossroads of scientific discovery,' said Venkata Indurthi, Chief Scientific Officer at Aldevron. 'This Innovation Center is a launchpad for transformative research, empowering us to innovate, collaborate and grow alongside the brightest minds in biotech.' The Waltham Innovation Center will complement Aldevron's established scientific teams in Fargo, ND, and Madison, WI, and will serve as a catalyst for recruiting top talent from Boston's vibrant biotech community. 'Our recent role in the groundbreaking 'Baby KJ' CRISPR therapy delivery demonstrates the life-changing impact that comes from investing in innovation,' said Jennifer Meade, President of Aldevron. 'As one of the most established biotech partners in the U.S., this expansion is a natural evolution—opening new opportunities for growth, deeper client partnerships and greater support for the scientists shaping the future of medicine.' About Aldevron Founded in 1998, Aldevron is a global leader in enabling the development of next-generation genomic medicines. As part of Danaher Corporation, Aldevron empowers scientists and innovators worldwide to advance transformative therapies that are redefining the future of medicine. Aldevron's expertise and integrated solutions have supported landmark achievements—including playing a key role in manufacturing the world's first mRNA-based personalized CRISPR therapy. With facilities in Fargo, ND, Madison, WI and Waltham, MA, Aldevron is at the forefront of accelerating scientific discovery and expanding the possibilities of gene editing, gene therapy and other breakthrough modalities. By partnering with leading researchers and organizations, Aldevron is helping to turn the promise of genomic medicine into reality for patients around the globe. To learn more about how Aldevron is advancing biological science, visit and follow the company on LinkedIn, Facebook and YouTube. ABOUT DANAHER Danaher is a leading global life sciences and diagnostics innovator, committed to accelerating the power of science and technology to improve human health. Our businesses partner closely with customers to solve many of the most important health challenges impacting patients around the world. Danaher's advanced science and technology - and proven ability to innovate - help enable faster, more accurate diagnoses and help reduce the time and cost needed to sustainably discover, develop and deliver life-changing therapies. Focused on scientific excellence, innovation and continuous improvement, our approximately 63,000 associates worldwide help ensure that Danaher is improving quality of life for billions of people today, while setting the foundation for a healthier, more sustainable tomorrow. Explore more at
