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.
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.
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.
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.
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.
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.
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.
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.
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.
A new CRISPR method, SEED/Harvest, has been developed to precisely modify DNA in fruit flies using the Single-Strand Annealing repair pathway. This allows for genome-wide changes with minimal unwanted scars.
Researchers identified nidogen-2 as a key driver of pancreatic cancer progression and metastasis. Blocking this molecule enhanced chemotherapy effectiveness and reduced spread in mouse models, suggesting a promising new treatment approach.
Researchers at the Arc Institute have discovered a novel bispecific guide RNA, the bridge recombinase mechanism, which enables precise and powerful tool to recombine and rearrange DNA in a programmable way. The system can insert any desirable genetic cargo into any genomic location with high efficiency and specificity.
Researchers at the University of Sydney have developed SeekRNA, a programmable tool that can precisely target and relocate genetic sequences with high accuracy and flexibility. This breakthrough technology surpasses current limitations of CRISPR, enabling more precise editing and reducing errors.
The study reveals a unique, ring-shaped organization of the antennal lobe, with specific glomerular clusters encoding different odors. This coding mechanism differs from other insects and vertebrates, with the representation of odor valence encoded in higher brain centers.
Researchers used CRISPR to fine-tune sugarcane's leaf angle, capturing more sunlight and increasing biomass production. The study focused on the LIGULELESS1 gene, which plays a major role in determining leaf angle.
This study successfully edits the rice genome with AsCas12f variants, achieving editing efficiencies of up to 53.1%. The research reveals unique deletion patterns primarily concentrated at positions 12-24, suggesting substantial potential for targeted DNA deletion using these miniature Cas12f variants.
Genome editing holds promise for molecular breeding, but delivery methods are hindered by tissue culture processes. RNA and DNA viruses have been employed to overcome these challenges. Geminiviruses offer a high copy number for delivering repair donors, while new vector systems and compact nuclease delivery are being explored.
A new study from Aarhus University has identified a gene that determines whether patients with prostate cancer develop metastases. The KMT2C gene is found to be crucial for the spread of prostate cancer and loss of this gene increases the risk of developing metastases.
A Japanese research team used machine-learning-driven modular assembly systems to create a more efficient gene editing tool. The study demonstrated an improvement in genome editing efficiency by 5%, showcasing the effectiveness of engineering zinc-finger nucleases through structural modeling.
A KAUST team developed a simple approach to tackle CRISPR's deletion issue by targeting error-prone DNA repair pathways. By modulating specific genes, they reduced large deletions while enhancing homology-directed repair efficiency.
Researchers used CRISPR/Cas9 DNA editing to insert genetic mutations found in humans with Tourette disorder into mouse embryos, creating a model to study the neurobiology and test new medications. The mice exhibited repetitive motor behaviors and sensorimotor gating deficits, similar to those seen in humans with the disorder.
The study, published in Cell Stem Cell, improves the growth of nephron progenitor cells (NPCs) using a chemical cocktail, enabling sustained growth in a simple 2-dimensional format. The breakthrough has potential for advancing kidney research and discovering new treatments.
A new LbCas12a variant, ttLbCas12a Ultra, achieved high editing efficiency in Arabidopsis, generating homozygous or biallelic mutants in a single generation. The authors optimized this variant for improved performance.
A new link has been discovered between FBXW7 mutations and EGFR signaling activity in colorectal cancer. The study found that the mutated form of the FBXW7 gene could no longer degrade the EGFR protein, leading to increased signaling activity and a decreased response to anti-EGFR treatment.
Tulane University researchers have developed a CRISPR-based platform for diagnosing nontuberculous mycobacteria (NTM) infections, allowing for accurate results in as little as two hours. The blood test can identify over 93% of patients with an NTM infection, enabling rapid treatment plans and reducing the risk of complications.
A new roadmap has been published by IEEE EMBS, outlining five primary medical challenges that need to be addressed through advanced biomedical engineering approaches. The paper, written by 50 renowned researchers from 34 prestigious universities, aims to guide future research and funding for groundbreaking innovations.
Researchers at CABBI developed a computational pipeline for identifying CRISPR/Cas-facilitated integration sites, which can pinpoint neutral integration sites in two to three minutes. This tool enables researchers to efficiently locate all the needles that align with their specific criteria, transforming the genome editing process.
Researchers at Durham University and Oxford Brookes University have identified a key gene contributing to the rapid evolution of male external genitalia in fruit flies. The study found that changes in the Sox21b gene altered genital shape and size, affecting mating duration and female choice.
Researchers have created a novel genome editing tool using zinc-finger DNA-binding domains, allowing for precise targeting and correction of disease-causing genetic mutations. This breakthrough enables safer and more adaptable gene therapy applications.
A new dual-function selection system enables both positive selection of multigene CRISPR mutants and negative selection of Cas9-free progeny in Arabidopsis. This system leverages a DAO-based surrogate selection marker to facilitate efficient multiplex CRISPR editing in plants.
Researchers at Tokyo Medical and Dental University develop a genome-editing technique that decreases PMP22 protein levels in patient cells, potentially reversing CMT-related changes. The study aims to improve myelination abilities and reduce symptoms in patients with CMT type 1A.
Gladstone scientists have created an intricate map of how the immune system functions, examining the detailed molecular structures governing human T cells. This study will accelerate the development of new and better therapies for cancer and autoimmune diseases.
Researchers found that certain combinations of gene mutations resulted in predictable effects on tomato size, while others yielded random outcomes. The study suggests the role of background mutations demands reassessment for genome editing applications. This new interpretation may help humanity adapt crops to meet evolving societal needs.
Researchers at Osaka University have developed a new gene editing technique called NICER, which significantly reduces off-target mutations compared to traditional CRISPR/Cas9 methods. This novel approach uses multiple small cuts in DNA strands and promotes interhomolog homologous recombination to correct heterozygous mutations.
North Carolina State University researchers successfully transferred an important gene from one compartment of a plant cell to another, producing tobacco plants that lack pollen and viable seeds. The findings could lead to better ways of producing hybrid seeds to maximize crop productivity.
A research group at Kyoto University has successfully developed a self-fertile buckwheat variety and a new type of the crop with a sticky texture. This breakthrough could contribute to the efficient breeding of less-common orphan crops, addressing the world's growing food demands.
Researchers successfully modified the ethylene synthesis pathway in the Japanese luxury melon to increase its shelf-life. The study found that introducing a mutation into the CmACO1 gene reduced ethylene generation, resulting in firmer fruit and longer shelf life.
Researchers from Tokyo Medical and Dental University successfully generated functional parathyroid glands from mouse embryonic stem cells using blastocyst complementation. This breakthrough study demonstrates the potential for regenerating organs in vivo and provides a new treatment option for hypoparathyroidism.