Articles tagged with Crisprs
Charité successfully treated 19-year-old Mohammad with CRISPR-based Exagamglogene Autotemcel, ending his dependence on blood transfusions. The treatment, which took 12 months, restored his ability to produce fetal hemoglobin, effectively eliminating oxygen deficiency in his blood.
Researchers have discovered a gene, Irx5, that enables blood cancer cells to evade treatment by boosting survival protein levels. By activating specific genes using CRISPR activation tool Partita, the team also identified new potential treatment targets for aggressive lymphoma.
Researchers at Tulane University have developed a diagnostic platform that can identify the precise species of bacteria causing NTM lung disease infections within four hours. The platform, called CANDI, uses mucus from the lungs and other respiratory fluids to test for up to 15 clinically relevant species and subspecies of NTM bacteria.
Researchers have developed a novel genome engineering method called prime assembly, which enables long DNA fragments to be stitched into precise target positions within living cells. This approach may allow for the development of universal gene therapies that can apply to many patients.
Scientists at Gladstone Institutes and UCSF discovered a pair of genetic edits that make CAR-T cells more effective against solid tumors. The new CRISPR screening platform enables the study of gene edits on CAR-T cells inside living mice, uncovering therapeutic targets previously missed in conventional cell culture systems.
Researchers created nearly 700 new cancer models derived from patient tumors to aid in drug development. The models, representing 25 types of cancer, are now available for global use, providing a resource for identifying new drug targets and testing potential treatments.
Researchers at the University of Illinois developed a gene editing tool to treat Huntington's disease by altering a specific point in the huntingtin gene. The treatment reduced toxic protein fragments, symptoms, and brain degeneration in mice, providing a new approach for treating genetic diseases.
Researchers found that CRISPR-Cas systems regulate the expression of innate immunity genes to defend against bacteriophages. This layered regulatory hierarchy provides a layer of protection, making it challenging for phages to evade the defense mechanisms.
Researchers used CRISPR and AI to identify two novel drug targets for psoriasis: the oxytocin receptor and ALOX5 enzyme. Topical gels containing these compounds reduced inflammation in mice as effectively as widely used injected therapies, offering a promising new approach to treating moderate-to-severe disease.
A new gene circuit technology has enabled cells to autonomously generate programmed responses, processing multiple molecular signals at once. The RATEX platform allows cells to compute and respond to various types of molecular information.
A genome editing technique called base editing has been used to study the role of a master gene in human embryonic cells, revealing its crucial function in early development. The technique allows scientists to alter a single gene in human embryos, enabling them to better understand how human embryos develop.
A large-scale Phase 3 trial of CRISPR therapy has shown an 87% reduction in attacks for patients with hereditary angioedema. The treatment also improved quality-of-life scores and reduced the need for on-demand medication, paving the way for future genetic therapies.
Researchers identified a promising new strategy for reversing autism-related brain deficits by targeting a specific glycine transporter. The therapy restored NMDA receptor function in mouse models and human brain organoids, improving behavioral abnormalities such as social interaction and repetitive behaviors.
The SMArT platform achieves near-pure selection of correctly edited blood stem cells while reducing dangerous genomic alterations. The innovative strategy enables enrichment of cells with targeted integration to 100% purity.
A breakthrough in CRISPR technology uses DNA to guide enzymes, overcoming the limitations of RNA-based systems. This new approach enables precise control over disease-causing signals, reducing unintended effects and costs, while opening doors to new treatment options.
Researchers discovered a molecular link between multicentric carpotarsal osteolysis (MCTO) and kidney disease, highlighting pathogenic MAFB accumulation and PI3K/AKT signaling. Treatment with imatinib suppressed AKT phosphorylation and attenuated glomerular injury in mice.
Researchers develop irreversible CRISPR base editing system to permanently block microbial survival, reducing environmental risk and genetic instability. The technology has broad applications in industrial biotechnology and biopharmaceutical fields.
Researchers summarize universal mechanisms of regulatory T cells in solid organ transplantation, enabling durable immune tolerance and reducing rejection. Gene-editing technologies create hypoimmunogenic Tregs for standardized, scalable availability.
Scientists discovered an anti-CRISPR protein that sabotages CRISPR systems in bacteria by jamming the protein assembly line. This discovery reveals a new mechanism of defense against CRISPR-based gene editing.
A new CRISPR tool has been developed to eliminate specific cells based on their RNA signature. The nuclease Cas12a2 can target any RNA sequence with high sensitivity and specificity, making it a promising approach for selectively killing cancer cells, virus-infected cells, and unmodified cells.
A new CRISPR protein, Cas12a2, has shown potential for killing sick cells while leaving healthy ones untouched. Researchers have tested its effectiveness in destroying cancer cells and virus-infected cells with promising results.
New technologies are shifting healthcare towards proactive care, using AI for early heart disease detection and miniaturized CRISPR-based diagnostics. These innovations enable seamless integration of existing medical screenings and provide laboratory-grade accuracy in a portable format.
Researchers used genome editing to inactivate a gene involved in anthocyanin production, resulting in increased accumulation of other flavonoids. This modification did not affect lettuce growth, suggesting a promising strategy for developing cultivars with tailored functional components.
Researchers at Gladstone Institutes identified hundreds of human genes influencing HIV infection and two potent antiviral proteins, PI16 and PPID. These proteins block HIV's entry into T cells or limit its ability to replicate within the cell.
A research team at HIRI discovered that CRISPR-Cas13 systems produce an RNA-based 'hairpin' structure to prevent the formation of waste RNAs, known as ecrRNAs. This mechanism optimizes the system's interaction and enhances its efficacy in immune defense and gene editing.
A team from The University of Osaka has identified the MIC11 gene as essential for parasite egress, disrupting the parasite life cycle. This finding could guide the development of novel treatments for parasite-borne diseases.
Researchers have discovered a smaller CRISPR enzyme, Al3Cas12f, that can efficiently edit genes in human cells. The enzyme's unique structure allows it to form a stable connection with DNA, making it a promising candidate for therapeutic genome editing.
The review emphasizes the need for robust non-clinical safety assessment to ensure CRISPR/Cas gene therapy products' safe translation. Central risks include genotoxicity and immunogenicity, mitigated by high-fidelity Cas variants and emerging delivery strategies.
Researchers at Bar-Ilan University have discovered that changing just one letter in DNA can completely alter sex development in mice. A single-letter insertion in a non-coding regulatory region caused XX mice to develop as males with testis and male genitalia.
Gladstone Institutes investigator Ryan Corces receives $750,000 to investigate unknown genetic variants contributing to Alzheimer's disease. He aims to identify new drivers and therapeutic targets using artificial intelligence and CRISPR tools.
Researchers have discovered an RNA-guided CRISPR system that can activate genes without cutting DNA, opening up new possibilities for gene regulation and therapeutic strategies. The system uses a strand of RNA as a guide to recruit the cell's transcription machinery, allowing for precise control over gene expression.
A genome-wide CRISPR/Cas9 knockout screen identified two critical host factors, SLC35A1 and LSM12, essential for BPIV3 replication. Knockout of these genes significantly inhibits viral infection, revealing novel antiviral strategies to combat Bovine Respiratory Disease Complex.
A new approach, called INSTALL, enables non-toxic DNA integration in multiple human cell types and successfully inserts large genetic payloads in mice, offering a promising solution for genetic therapies. The study's findings have the potential to broaden the applicability of genome editing therapies.
Engineers have refined a technology to edit individual genetic base pairs, reducing unintended edits and increasing safety for potential treatments. The new base editors could lead to better outcomes for some cystic fibrosis patients and more accurate models for drug testing.
Researchers used a virus-based CRISPR system to edit the gatekeeper enzyme HMGR in petunias and lettuce, unlocking natural metabolic control for enhanced aromatic compounds and health-promoting antioxidants. The result was more vigorous growth, stronger floral fragrance, and increased nutritional value.
Harlequin ichthyosis is caused by ABCA12 mutations leading to defective lipid transport and loss of skin barrier function. Management includes neonatal care, systemic retinoids, daily emollients, and keratolytics, with improved survival rates and quality of life.
A team of researchers uses CRISPR gene editing to eliminate cells with amplified oncogenes, reducing tumour growth and increasing animal survival. The study offers a promising approach for precision therapies in resistant cancers.
A field-deployable CRISPR-based biosensing platform has been developed for rapid, on-site monitoring of marine species and ecosystems, offering a sustainable solution for tracking ocean health. The technology has the potential to detect critical species, predict outbreaks, and support early warning systems for ecosystem disruptions.
Researchers at UC San Francisco have identified CUL5, a protein that tags tau for elimination, as a key player in preventing the formation of toxic tau protein clumps that can lead to dementia. The study found that neurons with more CUL5 are less vulnerable to Alzheimer's disease.
Researchers at the University of British Columbia have developed a topical CRISPR-based therapy that can correct faulty genes in human skin, potentially treating genetic skin conditions like ARCI and eczema. The treatment, using lipid nanoparticle technology, restores up to 30% of normal skin function.
Researchers at Monash University have developed an AI-powered approach to create highly accurate and specific anti-CRISPR molecules, enabling faster development of gene editing tools for various applications. This breakthrough addresses the inconsistent performance and safety risks associated with CRISPR technology.
Researchers discovered that CHD1 and MAP3K7 gene deletion improves tumor vulnerability to immunotherapy. This finding suggests new biomarkers for predicting patient response and opening up personalized cancer care. The study sheds light on why some patients are more or less likely to respond to certain types of cancer treatments.
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.
A new diagnostic platform enables rapid and accurate detection of drug-resistant C. auris pathogens using CRISPR technology, allowing for more effective treatment and prevention of hospital outbreaks. The dSHERLOCK test can detect the presence of mutations causing antimicrobial resistance in just 40 minutes.
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 discovered genes that regulate fibroblast growth, which builds the scaffolding between cells. Adjusting these factors reversed age-related changes and improved health outcomes in mice. The study offers new opportunities to understand and reverse aging-related diseases.
Researchers have identified a novel CRISPR mechanism, Cas12a3, that specifically targets transfer ribonucleic acids (tRNA) in bacteria. This discovery provides new insights into the immune response of bacteria and has potential applications for molecular diagnostics and other technologies.
A CRISPR screen identified 331 essential genes for brain cell generation, including PEDS1 linked to a severe developmental disorder. The study provides a new approach to identifying genes involved in neurodevelopmental disorders like autism and offers insights into gene inheritance patterns.
Researchers at Nara Institute of Science and Technology discovered that parasitic plants recognize
Researchers have identified DNA switches that control how brain cells called astrocytes work, which are known to play a role in Alzheimer's disease. The study used CRISPRi technology and single-cell RNA sequencing to test nearly 1000 potential switches, finding that about 150 of them controlled genes implicated in Alzheimer's disease.
Scientists at Penn Vet have identified two genes, Ctnna1 and Bcl2l13, that suppress metastasis in preclinical models of colorectal cancer. These findings could lead to better treatments and therapies for patients with metastatic disease.
Researchers used CRISPR to increase fungal production efficiency and cut environmental impact by 61% without adding foreign DNA. The genetically tweaked fungus tastes like meat and is easier to digest than its naturally occurring counterpart.
Researchers have shown that disabling the NRF2 gene with CRISPR technology can restore drug sensitivity and slow tumor growth in lung cancer. The approach, which targets a master switch for resistance, has potential across multiple tumor types.
Researchers found that CRISPR-Cas9 gene editing persists longer and produces more predictable results in non-dividing neurons. They also discovered new DNA repair genes that can be used to control gene editing outcomes, which could lead to safer and more effective therapies for genetic diseases.
A recent study published in Nature Communications reveals that the mechanical properties of the developing brain play a significant role in synapse formation and electrical signal emergence. The researchers found that softer regions exhibit higher synapse densities, while stiffer regions show lower densities.
Researchers at Gladstone Institutes and UCSF have identified the genetic switches that regulate FOXP3 levels in human and mouse cells. In humans, multiple enhancers work together to keep FOXP3 active, while a repressor keeps it off in conventional T cells. This discovery has important implications for developing immune therapies.
Researchers developed a scalable method to produce human kidney organoids, combining them with pig kidneys outside the body for transplantation. The transplanted organs functioned normally and showed no signs of damage or toxicity.
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 developed a new diagnostic test, CAARRD, to detect ribonucleic acid (RNA) at room temperature, increasing sensitivity and reducing complexity. The test uses special 'anti-tag' CRISPR sequences to block the activity of the Cas13a enzyme, allowing for faster and more affordable detection of viral RNAs such as HIV.
Researchers at CNIO have created a 'human repairome', a catalogue of 20,000 DNA 'scars' that reveal how genes affect DNA repair. This information can help determine the best treatment for each cancer type and overcome resistance to therapy.