A two-step genome editing method integrates large human genomic fragments into mice, mimicking human regulatory landscapes. This platform enables the creation of physiologically relevant humanized models for therapeutic targets and disease research.
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The CRISPR-Cas3 system has been shown to induce reliable and extensive deletions of the TTR gene in mouse models of ATTR, reducing serum TTR levels by up to 80%. This technology holds promise for treating not onlyATTR but also other incurable inherited diseases.
Researchers at Utah State University have discovered a new CRISPR system that can precisely target transfer RNA in invading pathogens, which could lead to the development of a single diagnostic test for COVID, influenza, and RSV. This discovery enables the detection and targeting of specific pathogens without damaging host cells.
Researchers use CRISPR/Cas9 to edit the mstnb gene in grass carp, resulting in denser muscle tissue with a higher number of fibers. This breakthrough allows for targeted and precise changes in muscle growth, promising improvements in aquaculture.
Researchers at Nara Institute of Science and Technology discovered that parasitic plants recognize
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Researchers at Cold Spring Harbor Laboratory use CRISPR to edit the goldenberry plant, reducing its growth by 35% and making it suitable for denser farming. The team hopes to breed plants with desirable traits such as fruit size and disease resistance.
Researchers used CRISPR technologies to discover previously unannotated DNA stretches in the 'dark genome', which control cell response to mechanical properties of their environment. This work could lead to new therapeutic targets for illnesses involving changes to tissue mechanics, including fibrosis and cancer.
A Mount Sinai study found that the timing of a genetic mutation in children with leukemia can significantly impact its aggressiveness. The researchers discovered that leukemia caused by mutations occurring before birth is often more aggressive and harder to treat than those occurring later in life.
A roundtable meeting in Morocco brought together experts to discuss precision breeding technologies for nutritional security and crop resilience. Precision breeding offers a targeted approach to improve crop genetics, addressing malnutrition and climate change impacts.
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Researchers discovered a 'sticky' molecule, P-selectin, that can cause blood clots and organ failure during COVID-19. A new mRNA therapy that drives P-selectin expression provides broad protection against coronavirus infection.
Researchers at the University of Texas at Austin have developed a novel gene-editing method that can correct multiple disease-causing mutations simultaneously. This approach uses bacterial retrons to protect the microbes from viral infection and has shown promising results in correcting scoliosis-causing mutations in zebrafish embryos.
Researchers identified key genes connected to cellular lipid metabolism that guide the precise release of cytotoxic granules in human NK and T cells. This discovery explains how immune cells work and sheds light on diseases caused by genetic defects.
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Researchers found that Agrobacterium's virulence is more effective in its natural two-chromosome state, but it grows faster and handles stress better when fused into a single chromosome. This study opens the door for optimizing its use as a crop improvement tool or devising new ways to protect crops vulnerable to crown galls.
The CityUHK team is developing two core therapeutic medicines using state-of-the-art DNA surgery technology to treat liver and cardiovascular genetic diseases. Their approach offers a durable and long-lasting solution, eliminating the need for repeated medications.
Researchers discovered that the functional splitting of transposon-derived RNAs drove the emergence of Type V CRISPR-Cas immunity. This innovation enabled the development of compact nucleases with flexible guide RNAs, offering design principles to create smaller and more versatile CRISPR tools.
Scientists have developed a new method to systematically discover genetic boosters for CAR T cells, a type of immune therapy. By knocking out genes that weaken CAR T cell function, researchers found a surprising genetic target: RHOG, which increases therapeutic potential when knocked out with CRISPR technology.
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Researchers at UC San Diego have created a new genetic editing approach that uses small nuclear RNA base editing, which can modify the genetic code with greater precision and safety than CRISPR. This method has the potential to treat various diseases, including neurodegenerative, cardiovascular, and immune disorders.
Researchers at MIT have developed a new approach to gene editing that reduces errors by up to 90%, making it a safer alternative for treating genetic diseases. The technique uses modified versions of the Cas9 enzyme to target specific DNA sequences, reducing off-target effects and increasing precision.
Researchers at the Max Delbrück Center have successfully used base editing to correct mutations that cause autosomal dominant polycystic kidney disease (ADPKD) in human and mouse cells. The technique shows promise in reducing liver cysts, a key symptom of the disease.
Researchers from Mass General Brigham developed a bespoke CRISPR-Cas9 gene-editing enzyme to correct the genetic error causing multisystemic smooth muscle dysfunction syndrome, a rare condition associated with stroke and death in childhood. The therapy extended survival four-fold in mouse models of MSMDS.
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A recent study by Michigan Medicine researchers has found that CRISPR-Cas9 forms immune memories in bacteria by boosting spacer acquisition when RNA levels are low. This discovery expands our understanding of how bacteria safeguard their immune memory and may inspire new ways to design CRISPR-based molecular recording tools.
Researchers at Cranfield University have developed a DNA-free gene editing technique for raspberries, which could lead to the creation of more sustainable and resilient varieties. The new method uses CRISPR-Cas9 technology to edit the genome of raspberry protoplasts, resulting in faster breeding times and reduced food waste.
Researchers have developed 'molecular scissors' that can precisely and permanently disable the hepatitis B virus's hidden genetic material. The treatment has shown promising results in laboratory tests and HBV-infected mice, with a 99% reduction in circulating viral DNA. This innovation represents a significant step towards a functiona...
A research team developed a new method to precisely edit DNA by combining genetic engineering with artificial intelligence. The technique enables accurate modeling of human diseases and lays the groundwork for next-generation gene therapies.
Researchers have developed novel methods to advance precise chromosomal manipulation by addressing challenges in the Cre-Lox system. Their innovations include asymmetric Lox site design and a protein-directed evolution system, enabling targeted integration of large DNA fragments up to 18.8 kb.
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Scientists successfully edited DNA directly in the brain to correct ultra-rare genetic mutations causing alternating hemiplegia of childhood. The technique improved symptoms and survival rates in mice, with implications for treating other rare genetic diseases.
Researchers used prime editing to correct five different AHC-causing genetic mutations in mice, resulting in far fewer and less severe symptoms. The treatment successfully repaired up to 90% of treated cells, demonstrating its potential for treating people with this rare neurological disorder.
Researchers at Rutgers University have discovered how specific sections of corn DNA control vital traits like plant architecture and pest resistance. The findings provide new insights for scientists to use innovative technologies to enhance corn crops.
A team of researchers from The University of Osaka has made a breakthrough in weight loss treatment by developing a one-time genome editing approach that introduces a GLP-1 receptor agonist gene. This innovative method enables the body to produce its own weight-loss medication, reducing the need for regular injections.
Researchers developed Variant-aware Cas-OFFinder, a web-based tool that improves CRISPR accuracy by identifying off-target effects across genetic variations. The tool offers a significant step forward in personalized genome editing by incorporating genetic diversity directly into off-target predictions.
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Frank Buchholz's ERC project DC-PGE aims to develop fully programmable DNA editing enzymes that minimize off-target effects and increase safety in gene therapy. The goal is to create a platform for efficient and accurate genome editing tools to treat various genetic disorders.
Researchers argue that deliberate full extinction might be acceptable in rare cases, but only with careful consideration of ecological and moral implications. The study calls for robust ethical safeguards and inclusive decision-making frameworks to guide the use of genetic modification technologies.
A new apomixis system termed Fix4 achieves stable and heritable clonal seeds with normal seed-setting rates, overcoming the limitations of previous genome editing systems. This innovation has significant implications for accelerating the application of apomixis technology in hybrid rice production.
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A team of researchers has successfully treated an infant with a life-threatening, incurable genetic disease using personalized gene editing therapy. The infant, who was diagnosed shortly after birth, showed positive responses to the treatment and improved symptoms over time.
The evoCAST system enables precise insertion of entire genes into the human genome, overcoming a major challenge in gene therapy. This breakthrough could lead to more reliable treatments for diseases like cystic fibrosis and hemophilia.
Spearhead Bio's TAHITI technology enables seamless integration of genes into crops, promising faster and cleaner path to crop improvement. The startup aims to generate next-generation improved crops with desired traits, improving speed to market and consumer acceptance.
Researchers at Karolinska Institutet have developed a technique to deliver gene editors and protein therapeutics to cells using engineered extracellular vesicles. The method shows promising results in animal studies, highlighting the potential for treating genetic diseases and neurological disorders.
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Common prescription medications can disrupt sterol biosynthesis, potentially causing developmental disorders. The editorial highlights the need for mandatory sterol biosynthesis screening in clinical practice.
Researchers use a new pipeline to make genetically engineered plants with improved oil production, reducing labor and time in the process. The FAST-PB platform integrates automation and single-cell lipidomics to accelerate plant transformation.
Scientists developed a new technology to produce Cre-loxP organisms in a single step, reducing the need for crossbreeding and decreasing production time. The method involves introducing a TAx9 sequence to prevent Cre gene expression in E. coli bacteria, allowing for precise control and modification of gene expression.
Scientists at CSHL and global collaborators have sequenced complete genomes for the Solanum genus, including tomatoes, potatoes, and eggplants. The study reveals the importance of understanding paralog genes in predicting genome editing outcomes.
Researchers successfully created a bi-paternal mouse by modifying genes involved in reproduction. The mice that reached adulthood exhibited altered growth and shortened lifespan, but could potentially lead to new therapeutic strategies for imprinting-related diseases.
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Researchers have successfully developed a gene-editing approach using CRISPR-Cas9 to correct the genetic error causing dysferlin protein deficiency, a leading cause of muscular dystrophy. In new mouse models, they restored muscle function and regrowth after transplanting corrected cells.
Five Texas researchers have been honored with the 2025 Edith and Peter O'Donnell Awards for their innovative breakthroughs in small cell lung cancer, lithium-ion battery technology, and galaxy discovery. Lauren Averett Byers is being recognized for her work on novel therapeutic strategies for SCLC, while Caitlin M. Casey is exploring p...
Researchers developed a non-transgenic genome editing approach in tobacco using an RNA virus vector, resulting in heritable edits and mutant lines with reduced nicotine content. The approach allowed for the simultaneous targeting of multiple genes involved in pyridine alkaloid biosynthesis.
Stanford Medicine researchers used a new gene-screening method to identify the CA12 gene as a key factor in doxorubicin-induced heart cell damage. They found that a drug called indisulam may be able to prevent this toxicity, and have tested it in mice with promising results.
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A team of Kobe University bioengineers successfully produced artepillin C in bioengineered yeast, achieving ten times the previous yield. The production process involved carefully tuning key steps along the molecular production line, and further improvements are being explored to increase efficiency.
The CRISPR tool was successfully used to correct a genetic defect in cells affected by chronic granulomatous disease. However, the repair process also introduced new genetic defects, highlighting the need for caution when using CRISPR technology in clinical settings.
A new study in mice shows a unique mRNA delivery method can successfully edit faulty genes in fetal brain cells. The technology has the potential to stop progression of genetic-based neurodevelopmental conditions like Angelman syndrome and Rett syndrome before birth.
Dr. Fyodor Urnav proposes a set of initiatives to address the crisis, including pooling patients by syndrome and permitting multiple gene editors in a single Investigational New Drug application. This approach aims to accelerate the development of CRISPR therapies for rare genetic diseases.
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Researchers at ChristianaCare Gene Editing Institute use CRISPR tools to safely disable gene mutation linked to treatment-resistant melanoma. The approach targets melanoma tumor cells while leaving healthy cells alone, restoring sensitivity to anticancer drugs.
Researchers developed a compact 'gene scissor' tool, TnpB, which shows a 4.4-fold increase in efficiency of modifying DNA, making it more effective as a gene editing tool. The tool can be used to treat patients with familial hypercholesterolemia, reducing cholesterol levels by nearly 80%.
Researchers found that fever temperatures increase helper T cell metabolism, proliferation and inflammatory activity, while causing mitochondrial stress, DNA damage and cell death in a specific subset of Th1 cells. These findings may explain how chronic inflammation contributes to cancer development and suggest a fundamental way cells ...
Scientists at Gladstone Institutes have discovered a diverse range of retrons that can edit DNA more quickly and efficiently than current methods, including CRISPR. The new retrons showed high editing rates in both bacteria and human cells, with some performing 10-fold better than the gold-standard retron.
Researchers at Osaka Metropolitan University used CRISPR/Cas9 to create a strain of Euglena that produces wax esters with shorter carbon chains, improving their cold flow and suitability as a biofuel feedstock. This breakthrough could potentially replace petroleum-based production of wax esters with biological sources.
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A new method called VitelloTag has been developed at the Marine Biological Laboratory, allowing researchers to deliver miniature research tools into egg cells and embryos. The approach uses a yolk protein found in most animals to bind to the receptor on the egg cell surface, enabling efficient delivery of CRISPR-Cas9.
A team of international researchers has discovered a surprising genetic mechanism that influences the vibrant patterns on butterfly wings. An RNA molecule controls where dark pigments are made during butterfly metamorphosis, shaping the butterfly's color patterns in a way previously unforeseen.
Researchers discovered a bacterial defense strategy involving two proteins that team up to disable plasmids, which could be applied to gene editing. Guide DNA and a functional protein are key components of this system, showing promise for targeted genome editing.
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Researchers found that brief, temporary changes to bacterial gene regulation imprint lasting changes within the network that are passed on to offspring. This discovery challenges long-held assumptions of how simple organisms transmit and inherit physical traits.
A new study reveals a connection between metabolic genes and immune system T cells, suggesting a potential new class of inborn errors of immunometabolism. Researchers identified genetic overlap between disorders of metabolism and immunity, pointing to a continuum between the two conditions.