CRISPR

Genome editing is not designed to replace neurons or retinal cells that have already been lost. In this article, experts from four companies—eGenesis, Epicrispr, KOMO Biosciences, and Sumitomo Pharma America—discuss how they are moving beyond conventional CRISPR to develop novel approaches for treating disease. The post Beyond Conventional CRISPR appeared first on GEN - Genetic Engineering and Biotechnology News.

A UCLA-led team has described how they advanced an experimental CRISPR approach for sickle cell disease into a blood stem cell therapy that can be manufactured at the scale and quality required for a clinical trial.

Some genetic diseases leave cells without a crucial protein. One possible treatment is to persuade genes already inside those cells to work harder, but the necessary machinery can be difficult to deliver, especially when researchers want to target several genes at once. A new compact CRISPR system devised by Hainan Zhang of the Shanghai Institute of Materia Medica, Chinese Academy of Sciences, and colleagues fits those instructions into a single viral delivery package.

The first patient received a dose of the high-precision gene editor in a new clinical trial for Wilson disease. Researchers hope it's a one-and-done fix. The post CRISPR Prime Editing Rewrites DNA in the Human Body for the First Time appeared first on SingularityHub.

Human cells hold about two meters (6.6 feet) of DNA, folded and compacted to fit inside a nucleus. That packing is essential for life, but it creates a problem for scientists. Some of the genes that control a cell's identity are buried so deep in tightly packed DNA that gene-targeting tools such as CRISPR activation, or CRISPRa, struggle to reach them.

The large language model took less than a day to identify the curious stretch of DNA

Biotechnology company QUiCKR Bio is using liquid handling solutions from INTEGRA Biosciences to run its CRISPR edit confirmation assays.

Scientists have long used a powerful gene-editing tool called CRISPR to make precise changes to DNA. But a different version of the technology, known as Cas13a, targets RNA, the genetic material found in viruses such as HIV.

A new engineered gene-editing system, proven in bacteria and showing promise for use in plants, animals and humans, addresses some limitations of current gene-editing tools, with the goal of improving their use in research, crop development and disease treatment.

INTEGRA's automated solutions optimize CRISPR workflows, enhancing gene editing efficiency and accelerating the path to innovative therapeutic developments.

A single dose of an experimental CRISPR therapy cut LDL cholesterol and triglycerides by about half in patients with difficult-to-treat lipid disorders. The reductions were still present one year later, with the highest dose lowering LDL by 52.5% and triglycerides by 47.8%.

A CRISPR-based method for disrupting genes across bacteriophage genomes has been developed, creating a faster route to uncover gene functions and engineer phages for potential therapeutic and biotechnology applications. The post New CRISPR-Based Tool Enables Genome-Wide Mutagenesis of Bacteriophages appeared first on GEN - Genetic Engineering and Biotechnology News.

Researchers used CRISPR to remove CD33 from donor stem cells, potentially giving doctors a way to attack aggressive blood cancers without destroying the healthy cells patients need after a transplant. In a 30-patient trial, the edited cells successfully took hold and appeared to shield blood cells from a CD33-targeted cancer treatment.

Anthropic says its Claude AI system identified a previously uncharacterised enzyme system in bacteriophage DNA, but researchers are still investigating whether it can perform programmable genetic operations.

Claude and the ART Enzyme System More than 200,000 enzymes went in, and one odd system came out.

Researchers have uncovered a previously unknown gene causing resistance to a leading blood cancer drug and several genes that accelerate lymphoma growth by using a powerful new CRISPR activation library.

CRISPR may be a powerful technology for gene editing, but the system existed in bacteria long before scientists began using it. For billions of years, CRISPR has acted as a natural immune system in bacteria and other microbes, helping defend them against invading viruses. But now, two new studies, published in Science, describe a similar RNA-guided system that originated in the viruses themselves and appears to be a precursor to CRISPR.

TIGRa, half CRISPR’s size, fits in viral vectors to boost protective genes. Its compact design could enable gene

Editing a plant's genes is only half the battle. Before a new, improved trait can ever reach a farmer's field, scientists must be able to grow a whole plant from just a few edited cells—a process that can be slow, unreliable and, for some plant species, impossible.

A research team from The Hong Kong University of Science and Technology (HKUST) has developed an innovative "one-pot" testing platform, known as TEMPO, that could transform highly sensitive nucleic acid testing from a laboratory-based procedure into a simple, single-step test that can be performed at home. With a single reaction tube, users can obtain results comparable to those of professional laboratory tests in as little as 30 minutes. The breakthrough has the potential to bridge the technological gap between existing rapid tests and laboratory-based nucleic acid diagnostics, offering a more convenient solution for future infectious disease surveillance and genetic screening.

The ability to precisely adjust the expression of genes inside the human body—ramping up protective ones and tamping down harmful ones—holds enormous potential for treating and preventing disease. But the standard molecular tools used to tune genes, such as the CRISPR system, are too bulky to package and deliver into the body's cells.

Like a delivery driver navigating crowded city streets, a gene-therapy-toting lipid nanoparticle faces a gauntlet of potential detours on its journey toward a cell's nucleus. First, there's entering the cell's plasma membrane; then navigating around organelles like the Golgi apparatus, mitochondria and endoplasmic reticulum—all destinations that can errantly absorb the particle's payload, rendering it ineffective at best or harmful at worst. And that's all before the particle even enters the nucleus and successfully makes a genetic change.

Like a delivery driver navigating crowded city streets, a gene-therapy-toting lipid nanoparticle faces a gauntlet of potential detours on its journey toward a cell's nucleus.

The puppies represent a key first step towards addressing canine allergenicity and mark potentially a significant advance in veterinary biotechnology. The post Fur Real: Biotech Startup Eliminates Major Dog Allergen in Beagle Pups Using CRISPR appeared first on GEN - Genetic Engineering and Biotechnology News.

Scientists used CRISPR to make prostate cancer cells easier for the immune system to detect and destroy. The experimental treatment dramatically improved the effects of immunotherapy in mice and may offer hope for other hard-to-treat tumors.

The main function of CRISPR-Cas systems, the revolutionary tool scientists now employ to edit genes, is to defend bacteria against threats such as viruses called bacteriophages.

To make CRISPR better at its job, researchers are turning to algorithms like DeepMind's AlphaFold. The post Scientists Are Designing CRISPR Gene Editors With AI appeared first on SingularityHub.

All cellular life-from humans to bacteria-possesses two layers of immunity: innate and adaptive. Scientists have long understood how these two types of immunity work together in higher organisms but not in the microbial world.

All cellular life—from humans to bacteria—possesses two layers of immunity: innate and adaptive. Scientists have long understood how these two types of immunity work together in higher organisms but not in the microbial world.

UC San Diego has joined the Innovative Genomics Institute as its fourth UC partner, expanding collaboration to develop next-generation genome-editing technologies that address major health, climate, agriculture, and environmental challenges through interdisciplinary research. The post UCSD, IGI Partnership Expands CRISPR Innovation Across Health, Agriculture, and the Environment appeared first on GEN - Genetic Engineering and Biotechnology News.

Scientists have made many advances using traditional CRISPR technology, especially in medicine, but they are now seeking ways to create genuinely new gene-editing enzymes with properties that have not already evolved naturally. A new study, published in Science, describes a new AI-designed synthetic TnpB enzyme, called SynTnpBs, that has outperformed the natural reference enzyme.

Scientists used artificial intelligence to design CRISPR nucleases with properties not found in nature, achieving activity that matches or exceeds natural enzymes despite the proteins' complex, multi-domain structure. The post AI-Designed Synthetic CRISPR-Like Nucleases Show Activity in Cells appeared first on GEN - Genetic Engineering and Biotechnology News.

Combining CRISPR/Cas9 with CAR T aims to let patients’ T cells be reprogrammed in vivo, potentially lowering cost and expanding use to solid tumors and autoimmune disease.

The speed at which a cell produces proteins is a decisive factor in determining whether it divides, specializes or retains its stem cell properties. A team of researchers led by Professor Stefan H. Stricker, professor of epigenetic engineering at LMU's Biomedical Center and research group leader at Helmholtz Munich, has worked with international partners to demonstrate directly for the first time that the amount of ribosomal RNA (rRNA) directly regulates these processes. Their results were published in the journal Science.

In a study published in Science Translational Medicine, a team of researchers led by Dr. Wang Yu from the Shenzhen Institutes of Advanced Technology of the Chinese Academy of Sciences developed PRINCE and Little Prince, dual small-molecule-controlled genome editing systems that allow CRISPR activity to be switched on by drug inducers and kept largely silent in their absence.

Successfully treating Baby KJ with a personalized CRISPR gene-editing therapy is spurring the industry to investigate how to develop standardized manufacturing platforms as well as how individualized gene-editing products will be regulated. The post Standardizing Personalized CRISPR Gene-Editing Therapies appeared first on GEN - Genetic Engineering and Biotechnology News.

When standard leukaemia treatments failed, 13-year-old Alyssa Tapley was told she had only weeks left – but then she was offered an experimental procedure

A handful of start-up firms are testing therapies that target specific epigenetic markers to treat everything from high cholesterol to a rare muscular disorder

This GEN Live show will bring together a panel of leading experts to break down the latest advances, innovations, and challenges shaping genome editing. The post Genome Editing at the Turning Point—Bringing CRISPR to Clinical Reality appeared first on GEN - Genetic Engineering and Biotechnology News.

At present, more than 700 million people live with caloric hunger, and more than 2 billion suffer from micronutrient deficiencies, known as "hidden hunger." By prioritizing high yield over nutritional quality, global calorie production has increased while exacerbating vitamin and mineral deficiencies. Stress from climate change has been shown to further reduce the densities of several nutrients.

In a rapidly changing climate landscape, the plants we rely on for food, textiles and more face a multitude of challenges, including rising temperatures, drought and disease. Caltech's Gözde Demirer, the Clare Boothe Luce Assistant Professor of Chemical Engineering, uses genetic engineering tools to make crops more resilient to such threats and enhance plant health. Now, she and a team of Caltech researchers have found a new solution to an old problem in an unlikely source: the zebra finch.

A team of researchers led by Professor Akitsu Hotta (Department of Clinical Application) developed a comprehensive framework that combines computational prediction, experimental validation and whole-genome analysis to evaluate intended and unintended mutations arising from CRISPR-Cas9 delivered by lipid nanoparticles (LNPs), providing a practical strategy to improve the safety of genome-editing therapies. The work is published in the journal Molecular Therapy Nucleic Acids.

Researchers from Amsterdam UMC, in collaboration with other hospitals, have successfully completed the first-ever Phase 3 study of an in vivo CRISPR therapy.

Researchers from Skoltech—a VEB.RF group institution—and their colleagues from the U.S. and China have explained how the antibiotic resistance gene established itself in the genome of the bacterium Klebsiella pneumoniae. The findings could help control this widespread microbe, which can cause pneumonia, meningitis and other, often hospital-acquired, infections in patients with weakened immunity. The findings were reported in the Proceedings of the National Academy of Sciences.

“Guardian of the genome,” p53 is now therapeutically accessible using CRISPR-based technology from Jennifer Doudna’s lab. The approach uses RNA signatures to identify and destroy traditionally undruggable cancer cells. The post CRISPR Shreds Undruggable Cancer Cells with Precision appeared first on GEN - Genetic Engineering and Biotechnology News.

A team of researchers led by Luigi Naldini at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) has developed a new strategy to significantly improve the precision and safety of CRISPR-Cas9 gene editing in human blood stem cells, potentially overcoming one of the major barriers limiting broader clinical application of genome editing therapies.

Researchers at Cornell University have developed a safer and more precise way to study how genes function in living tissues by refining a recently developed CRISPR-based genetic technique in fruit flies, enabling researchers to better study how genes contribute to development and disease.

Researchers developed ΨDNA, a DNA-based CRISPR guide that allows Cas12 enzymes to target RNA with high specificity while retaining compatibility with DNA-editing workflows. The platform detected HCV RNA in clinical samples and enabled programmable RNA knockdown, multiplex targeting, and RNA modification in human cells.

A team of engineers at the University of Florida has developed a new form of CRISPR technology that could make diagnostics and treatments safer, more precise, and more affordable, while opening the door to entirely new ways of controlling disease.

The platform, called ΨDNA, reprograms Cas12 nucleases to recognize and act on RNA using a DNA-based guide scaffold. In human cell lines, ΨDNA achieved 70–95% knockdown of endogenous RNA transcripts. The post DNA‑Guided CRISPR Suggests a New Direction for RNA Editing appeared first on GEN - Genetic Engineering and Biotechnology News.

A team of engineers at the University of Florida has developed a new form of CRISPR technology that could make diagnostics and treatments safer, more precise, and more affordable, while opening the door to entirely new ways of controlling disease.

A Phase I/II clinical trial shows that a stem cell transplant that removes CD33 from donor cells using CRISPR can prevent cancer recurrence. The post Acute Myeloid Leukemia Therapy Improved by CRISPR Stem Cell Transplant appeared first on GEN - Genetic Engineering and Biotechnology News.

An enzymatic scissors recognizes and cuts viral RNA, but leaves the host cell unharmed. This opens up new antiviral strategies against hepatitis E.

Engineered microorganisms are widely used in industrial biotechnology and biopharmaceutical applications, including the production of biofuels, sustainable chemicals, and therapeutic compounds. However, concerns remain regarding the unintended environmental release and uncontrolled proliferation of genetically engineered microbes. For this reason, biocontainment technologies, which are designed to prevent microorganisms from surviving outside controlled environments, have become increasingly important in both academia and industry.

Bacteria fend off invading viruses with molecular scissors that slice up viral DNA—a system called CRISPR that's become indispensable to gene editing. But viruses can fight back with a molecular trick that stops the scissors from ever being made.

Among the challenges in treating disease, including cancer, is wiping out malignancies, infection, contaminants or other pathologies, without destroying healthy tissue.

A research team led by Prof. Hsing I-Ming, Professor of the Department of Chemical and Biological Engineering (CBE) at The Hong Kong University of Science and Technology (HKUST), in collaboration with Prof. Zhai Yuanliang, Associate Professor of the Division of Life Science (LIFS), has successfully developed the world's first DNA-guided CRISPR-Cas system capable of programmable RNA targeting and cleavage.

A new kind of CRISPR that destroys cells rather than gene editing them has shown potential for killing sick cells while leaving healthy cells untouched. The technology has largely been tested in cells in a dish, but if it can be applied to organisms, it could be a powerful tool to treat disease and advance research.

In this , Tilmann Buerckstuemmer, PhD, CSO at Myllia Biotechnology will show how high-throughput pooled CRISPR screening combined with cell painting readouts characterized important signaling pathways using NF-κB nuclear translocation as a case study. The post Optical Pooled CRISPR Screen Reveals Regulators of NF-κB Dynamics in Human Cells appeared first on GEN - Genetic Engineering and Biotechnology News.

As the spread of infectious diseases accelerates, technologies that can accurately distinguish multiple viruses in a single test are becoming increasingly important. KAIST and an international research team have developed a new diagnostic technology that simultaneously identifies various viruses and variants by controlling the "speed" of gene scissors.

JMIR Publications today featured two new stories in its News and Perspectives section, highlighting a shift toward proactive, tech-integrated health monitoring.

For most of their lives, plants get their energy from photosynthesis. But during the seed to seedling stage, when they can't absorb light just yet, they rely on other sources, like fatty acids. To process the fatty acids, plant cells, like human cells, rely on a membrane-bound compartment called the peroxisome. For people interested in studying the peroxisome, plant cells are an excellent model to use.

By applying base editing, scientists repaired a cystic fibrosis mutation that is unresponsive to current drugs in cell and organoid models, pointing to a possible treatment for some patients. The post CRISPR Base Editing Repairs Hard-to-Treat Cystic Fibrosis Mutation in Cell Models appeared first on GEN - Genetic Engineering and Biotechnology News.

Nature is the foremost international weekly scientific journal in the world and is the flagship journal for Nature Portfolio. It publishes the finest peer-reviewed research in all fields of science and technology on the basis of its originality, importance, interdisciplinary interest, timeliness, accessibility, elegance and surprising conclusions. Nature publishes landmark papers, award winning news, leading comment and expert opinion on important, topical scientific news and events that enable readers to share the latest discoveries in science and evolve the discussion amongst the global scientific community.

Using genome‑wide CRISPR activation and knockout screens in primary human T cells, researchers mapped the human genes that shape HIV infection and identified potent antiviral factors, including PI16 and PPID, with distinct mechanisms of action. The post CRISPR Screens Map Human T‑Cell Genes That Promote or Block HIV Infection appeared first on GEN - Genetic Engineering and Biotechnology News.

Cancer cells excel at evading detection, but subtle chemical differences set them apart from healthy cells. Now, a team of scientists from Wageningen University & Research and Van Andel Institute has identified a way to exploit this distinction. Using a variant of CRISPR, a modern tool for editing DNA, they distinguished tumor DNA from healthy DNA and selectively cut only the former. The study, published today in Nature, is an early but promising step toward a cancer therapy that targets and destroys tumor cells with high precision.

Dr. Swee Lay Thein and Dr. Stuart Orkin won the $3 million Breakthrough Prize in Life Sciences for their work toward a functional cure for the deadly blood disorders sickle cell disease and beta thalassemia.

Cancer cells excel at evading detection, but subtle chemical differences set them apart from healthy cells. Now, a team of scientists from Wageningen University & Research and Van Andel Institute has identified a way to exploit this distinction. Using a variant of CRISPR, a modern tool for editing DNA, they distinguished tumor DNA from healthy DNA and selectively cut only the former.

Cancer cells excel at evading detection, but subtle chemical differences set them apart from healthy cells.

A newly characterized Cas12f nuclease shows strong editing in human cells. Researchers engineered a variant with markedly improved efficiency, advancing efforts toward compact genome editors suitable for targeted delivery. The post Engineered Miniature CRISPR Boosts Gene‑Editing Efficiency in Human Cells appeared first on GEN - Genetic Engineering and Biotechnology News.

The CRISPR-Cas gene-editing system has long been the focus of research as a promising tool in genome editing. However, the emphasis has been on its underlying mechanisms and nucleases. In contrast, little research has examined how CRISPR-Cas systems have evolved and been optimized. In collaboration with the universities of Leipzig, Freiburg, and Michigan (U.S.), a research team at the Helmholtz Institute for RNA-based Infection Research (HIRI) in Würzburg found an optimization mechanism in CRISPR-Cas13, providing insights into the evolution of these systems. The results were recently published in The EMBO Journal.

A research team has discovered an enhanced CRISPR gene-editing system that could enable targeted delivery inside the human body—a key step toward broader clinical use. Researchers identified a naturally occurring enzyme, Al3Cas12f, that is small enough to fit into adeno-associated virus vectors, a leading targeted delivery method for gene therapies. They then engineered an enhanced version that dramatically improved gene-editing performance in human cells.

The rapid evolution of CRISPR/Cas genome editing has redefined the possibilities of cellular and gene therapy, enabling precise correction, disruption, and regulation of disease-associated genes.

An AI system unearthed a trove of CRISPR-like proteins in minutes instead of weeks or months. The post MIT Mined Bacteria for the Next CRISPR—and Found Hundreds of Potential New Tools appeared first on SingularityHub.

In the first of a new series of Keynote Webinars, Professor Rodolphe Barrangou, PhD (North Carolina State; EIC, The CRISPR Journal) offers a front-row perspective of the CRISPR revolution, the seminal advances, clinical highlights, and rising applications. The post CRISPR at 25: The Past, Present, and Future of Genome Editing appeared first on GEN - Genetic Engineering and Biotechnology News.

In back-to-back studies published in Nature, researchers from Purdue University and Columbia University report a naturally evolved gene-editing system that can activate genes, offering an advantage over existing CRISPR gene-editing systems that merely find and cut DNA. The research includes two complementary studies, one examining the biological function of the system and the other revealing the molecular mechanism that enables it.

Mechanistic studies suggest the amino acid cocktail enhances a clathrin‑independent, carrier‑mediated endocytic pathway, increasing the efficiency with which cells internalize LNPs. The post Amino Acid Cocktail Supercharges LNP Delivery for mRNA and CRISPR Therapies appeared first on GEN - Genetic Engineering and Biotechnology News.

Lipid nanoparticles, or LNPs, best known as the delivery vehicle for the COVID-19 mRNA vaccines received by billions of people, are now at the center of a much larger medical revolution.

Lipid nanoparticles, or LNPs, best known as the delivery vehicle for the COVID-19 mRNA vaccines received by billions of people, are now at the center of a much larger medical revolution. Researchers are racing to use them to ferry therapeutic mRNA into cells for cancer therapies and treatments for inflammatory diseases, as well as delivering CRISPR constructs that can correct disease-causing gene mutations.

After a heart attack, the heart struggles to recoup and maintain energy. One-third of patients develop heart failure as a result—a condition that impacts 6.8 million Americans and carries a high lifetime risk, with 1 in 4 adults in the U.S. expected to develop the condition during their lifetime. This makes finding lasting treatment a medical priority.

After a heart attack, the heart struggles to recoup and maintain energy. One third of patients develop heart failure as a result ⎯ a condition that impacts 6.8 million Americans and carries a high lifetime risk with 1 in 4 adults in the U.S. expected to develop the condition during their lifetime.