Articles tagged with Crisprs
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.
Researchers identified genetic modifications that can improve the efficacy of chimeric antigen receptor (CAR)-T cell treatment for multiple myeloma and other cancers. The study used CRISPR screening to pinpoint genes that influenced T cell function and survival in culture and in a preclinical model of multiple myeloma.
A new high-throughput screening approach has enhanced CRISPR genome-editing efficiency by identifying promising CAST variants. The method allows for rapid optimization of these candidates, uncovering mechanistic insights that can inform further engineering and potential clinical use.
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.
Scientists at UCSF successfully used CRISPRa to increase SCN2A levels in mice with the genetic disorder, resulting in reduced seizures and improved brain function. The therapy offers hope for treating neurodevelopmental issues related to SCN2A haploinsufficiency.
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 Stanford Medicine have created an AI-powered CRISPR tool called CRISPR-GPT, which helps scientists design and analyze gene-editing experiments. The technology aims to produce lifesaving drugs faster and expand access to gene editing for a wider range of scientists, including those with limited experience.
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.
Researchers at Northwestern University have developed a new CRISPR delivery system that triples efficiency using DNA-wrapped nanoparticles, improving safety and effectiveness. The new system, called LNP-SNAs, targets specific cells and tissues, reducing toxicity and boosting gene-editing efficiency by threefold.
A key contributor is the lab of Prof. Ayal Hendel at Bar-Ilan University's Goodman Faculty of Life Sciences, focusing on evaluating CRISPR-based gene editing precision and safety. The project aims to decentralize CAR-T cell therapy production, making it available to more patients by reducing costs and increasing accessibility.
Researchers have developed a new epigenetic editing method using CRISPR technology, which can switch genes back on by removing methyl groups attached to silenced or suppressed genes. This approach shows promise for treating people with Sickle Cell-related diseases, reducing the risk of unwanted changes and potential health problems.
Researchers at UC Berkeley developed a method to understand the isotope signatures of methane from different environments using CRISPR. This study can change how scientists calculate the contributions of various environmental sources to Earth's total methane budget.
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 at the Salk Institute have identified dozens of microproteins that play a crucial role in regulating fat cell proliferation and lipid accumulation. This breakthrough discovery offers new potential drug targets for treating obesity and metabolic disorders, building on recent advances in CRISPR gene editing technologies.
A team of scientists proposes an integrated framework combining biotechnology and AI to revolutionize crop breeding, exploring multi-omics, genome editing, and high-throughput phenotyping. The authors present a forward-looking framework for AI-assisted crop germplasm design, offering a roadmap for sustainable agriculture.
Researchers developed a CRISPR-based gene-editing system that changes a single molecule within mosquitoes, halting malaria-parasite transmission. The new system is designed to genetically spread the malaria resistance trait until entire populations of the insects no longer transfer the disease-causing parasites.
A team of scientists proposes using gene editing to restore lost genetic diversity in endangered species, enabling them to adapt to future environmental changes. The approach could complement traditional conservation methods and attract new investors and expertise.
Researchers successfully modulate the activity of key immune system genes using CRISPR-Cas9 technology, revealing a new approach to treating inflammatory diseases. The study demonstrates precise control over gene expression and its impact on tumour growth and inflammation.
Scientists have identified a set of genes in zebrafish that reactivate after damage to the heart and patch it up like new. The researchers hope to use CRISPR tools to reactivate similar genes in humans and jump-start repair of the heart and other tissues after injury.
The Center will develop personalized CRISPR on-demand treatments for children with severe inborn errors of immunity and metabolic disease. The initiative aims to make CRISPR cures more affordable and accessible, building on recent clinical success in treating ultra-rare genetic diseases.
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.
Researchers have successfully edited harmful mitochondrial DNA mutations in liver and skin cells using a genetic tool called a base editor. The study, published in PLOS Biology, offers promising results for treating mitochondrial diseases and aging-related conditions.
Researchers developed a new laboratory method that helps diagnose patients with suspected immune system disorders like activated-PI3Kδ syndrome (APDS). The method identified dozens of additional genetic variations associated with the condition, enabling rapid diagnosis and treatment. This breakthrough may also help identify more patien...
Researchers have found a promising new method for gene therapy by bringing dormant genes closer to enhancer switches on the DNA. This 'delete-to-recruit' strategy has potential for treating genetic diseases such as sickle cell disease and beta-thalassemia, offering an alternative to expensive current treatments.
Researchers at KAIST have developed a groundbreaking technology capable of selectively acetylating specific RNA molecules within the human body using the CRISPR-Cas13 system. This breakthrough enables precise, programmable control of RNA function and is expected to open new avenues in RNA-based therapeutic development.
Researchers leveraged CRISPR/CasRx to silence Ctnnb1 and Smo, two genes involved in osteoarthritis development. Simultaneous knockdown of both genes effectively mitigated joint structure degeneration, highlighting the potential of RNA-based gene therapies for OA management.
Researchers discovered that a ligase called Lig3 inhibits base editing, while the mismatch repair pathway helps cytosine base editing. The study sheds light on the complex mechanisms behind base editing and its potential applications in treating genetic diseases.
Researchers at Stanford University have developed a new CRISPR technology called CRISPR-TO that can transport RNA molecules to specific locations within neurons, enabling repair and regeneration. The technology has shown promising results in increasing neurite growth by up to 50% in mouse brain neurons.
Researchers at MSK uncovered a key signaling molecule involved in the body's immune response against leptomeningeal metastasis. A new grading system to assess thrombocytopenia risk after CAR T cell therapy was also developed. Additionally, a statistical method called UnitedMet estimates metabolic characteristics from challenging clinic...
Scientists at Rockefeller University have identified a novel CARF effector called Cat1 that prevents viral replication by depleting NAD+ metabolites. The discovery sheds new light on the complex molecular mechanisms behind CRISPR-Cas9 defense systems.
A new generative AI technique allows for the design of RNA molecules with improved functions, opening up potential for novel therapeutics and diagnostics. The SANDSTORM and GARDN systems enable the prediction and generation of RNA sequences tailored for specific tasks in cells or diagnostic assays.
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.
Mass General Brigham researchers developed a machine learning algorithm, PAMmla, to predict properties of genome editing enzymes. The approach helps reduce off-target effects and improves editing safety and efficiency, enabling customized enzymes for new therapeutic targets.
Tulane University scientists developed a handheld device to deliver rapid and accurate tuberculosis diagnoses in under an hour. The device, called the lab-in-tube assay (LIT), can detect Mycobacterium tuberculosis DNA in saliva, blood, and sputum samples, offering a cost-effective tool for improving TB diagnoses in resource-limited areas.
Researchers at KAIST discovered that DDX54 is the master regulator hindering immunotherapy's effectiveness in lung cancer. Supressing DDX54 enhances immune cell infiltration into tumors and improves immunotherapy efficacy.
Researchers have discovered a protective cloaking mechanism in jumbo phages that shield their genetic material from the host's immune system. This innovation could lead to new therapies for antibiotic-resistant infections.
Researchers developed a CRISPR-based diagnostic test that rapidly detects low levels of pathogen genetic material in blood without nucleic acid amplification. The test demonstrated unprecedented sensitivity and could be used to develop highly sensitive CRISPR-based diagnostic tests for detecting pathogens in minutes.
Researchers have discovered new CRISPR-Cas systems with improved efficiency and specificity, including one from dairy cow bacteria that can target specific gene sequences. The new systems have potential applications in human health, biotechnology and environmental fields.
A new method for detecting antibiotic resistance genes (ARGs) in wastewater has been developed by researchers, which uses CRISPR-Cas9 technology to enrich ARG fragments and increase detection sensitivity. This enhanced method was found to detect 1189 more ARGs and 61 more ARG families compared to standard metagenomics methods.
A breakthrough CRISPR-based test has been developed to diagnose Pneumocystis jirovecii pneumonia (PJP), a life-threatening fungal infection. The test detects RNA from live fungi in blood samples and throat swabs, providing a faster and more accurate diagnosis than current invasive bronchoscopy procedures.
Researchers use CRISPR-Cas9 to remove duplicate chromosomes in trisomy 21 cells, restoring gene expression and cellular phenotypes. The technique shows promise as a potential medical intervention for people with Down syndrome.
Researchers have discovered a new mechanism by which bacteria defend against CRISPR-Cas systems, and how phages counter these defenses. This discovery holds potential to enhance the safety and precision of CRISPR-based technologies.
Researchers discovered 47,350 active putative enhancers associated with Parkinson's disease, schizophrenia, and other neurological disorders. These enhancers were found to regulate gene expression during neuronal differentiation.
Scientists at Duke University have discovered a master epigenetic switch that can be activated using CRISPR to compensate for missing genes in Prader-Willi syndrome. This approach could potentially treat the disease by turning on naturally suppressed genes from one parent, addressing the underlying genetic defect.
Researchers used CRISPR/Cas9 to study the gene function of adenine phosphoribosyl transferase in beans. They found two functional mutants with distinct roles: one affects adenine recycling and the other regulates cytokinins, essential for root growth and nodules.
Researchers used CRISPR/Cas to generate controlled chromosomal inversions in Arabidopsis thaliana, finding no significant changes in epigenetic marks or gene expression. Only minor genome-wide effects were observed, indicating the robustness of the epigenome and transcriptome.
Researchers from the University of Lausanne used genome editing to repair a deleterious domestication mutation in the tomato genome. This resulted in an earlier yielding variety, which could have implications for agriculture and sustainability. The study demonstrates the potential benefits of genome editing for crop breeding.
Researchers at Gladstone Institutes and UCSF identified MED12 as a crucial switch that regulates T cell rest and activation. The study found that MED12 promotes rest in resting cells and activation in activated cells, and its removal led to blurred lines between rest and activation.
Researchers have discovered a major setback in the use of AZD7648 to promote precise gene editing, which causes massive genetic changes and genome instability. Despite this, scientists remain optimistic about advancing CRISPR-Cas technology to treat diseases.
Researchers at the University of Tokyo have developed a new CRISPR-based system to label small extracellular vesicles (sEVs) with RNA barcodes, enabling comprehensive analysis of their biogenesis and release regulators. This system allows for the simultaneous study of thousands of genes and estimation of sEV release from host cells.
Researchers have developed a new CRISPR-Cas method to decipher the function of genetic variants that contribute to cancer. The approach creates tens of thousands of cells with different gene variants, allowing scientists to identify which variants make cancer cells resistant to standard drugs.
A new study uses CRISPR-Cas13 to identify nearly 800 noncoding RNAs that are functional and essential for cell function, including in cancer and human development. The researchers found that these RNA molecules modulate key pathways for cell proliferation and can serve as potential biomarkers and therapeutic targets for cancer treatment.
Researchers have discovered a new type of CRISPR chemistry that floods infected cells with toxic molecules and shuts down activity, preventing viruses from spreading. The discovery sheds light on the complex mechanisms of CRISPR systems and their potential applications as diagnostic tools for infection.
A CRISPR-Cas9 based gene editing therapy has been shown to reduce angioedema attacks and sustainably lower kallikrein levels in patients with hereditary angioedema, offering a potential treatment option. The therapy was tested on 27 patients across two dosages compared to placebo.
A team of researchers has developed strategies to identify regulators of intestinal hormone secretion, which could lead to new treatments for metabolic and gut motility disorders. They used human organoids to study the function of 'nutrient sensors' on hormone-producing cells in the gut.
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.
Scientists at St. Jude Children's Research Hospital studied the structure of Fanzor2, a eukaryotic genome-editing protein, to understand its potential for gene editing. The findings reveal that Fanzor2 has a unique RNA-guided nuclease system, which could be harnessed to create more functional and smaller proteins.
The CRISPR-Cas13 system enables temporary gene expression manipulation without permanent genomic changes, holding promise for treating diseases caused by RNA defects. It has been applied to correct mutations linked to Duchenne muscular dystrophy and can be used to alter splicing events, making it a powerful tool in personalized medicine.
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%.