Articles tagged with Genome Editing
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 discovered that CRISPR-Cas plays a broader role in bacteria, directing a network of innate immune systems. Over 20 innate defense modules are genomically embedded within type I CRISPR-Cas loci, which are kept under tight transcriptional control by small RNAs.
Researchers use CRISPR-Cas9 to edit red perilla, producing green plants with increased levels of antioxidants and anti-inflammatory compounds. The study demonstrates a new strategy for developing high-value crops for the food and pharmaceutical industries.
Researchers identified a precise gene edit that lowers cadmium in rice grains while maintaining yield and essential mineral nutrients. The OsNramp5 I441T mutation selectively limits cadmium translocation, reducing grain cadmium by 48% without compromising zinc or manganese uptake.
Scientists identified 81 previously unrecognized cancer-driving genes in basal-like breast cancer, a subset with poor outcomes. The discovery expands understanding of chromosomal rearrangements and opens possibilities for targeted therapy.
A new strategy makes stem cell transplants safer by replacing chemotherapy-based treatment with targeted antibodies that recognize specific markers on blood-forming stem cells. This approach allows for less toxic treatment and could enable chemotherapy-free or chemotherapy-sparing transplantation approaches.
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 new Europe-wide survey found broad public support for fertility treatment and reproductive research in the UK, Netherlands, Spain, and Italy. The study suggests that public understanding and opinion on these topics continue to develop, with areas where public views are not yet settled identified.
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.
Researchers will develop genetic boosters and "two-factor authentication" for CAR T cell therapy, targeting HER2-positive lung, breast, and colon tumors. The project aims to improve the efficacy of CAR T cells against solid tumors while reducing collateral damage.
The US retains its scientific edge but is losing the race to translate discoveries into cures, warns a new report from Cure Innovation Index. Without immediate renewed investment and policy changes, the US scientific edge will not hold.
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 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 have successfully developed a new CRISPR system, Cas12a2, that can selectively kill cancer cells without harming healthy tissue. The technology uses a guide RNA to target specific genetic mutations and has shown promise in reducing tumor volume by 50% after a single treatment.
A new technology allows for the efficient insertion of large DNA segments, enabling a 'chapter rewrite' in the genome. This method avoids double-strand breaks and can correct hundreds of mutations simultaneously.
Researchers have developed a universal toolkit for editing bacterial DNA in 15 diverse species, including human pathogens and fast-growing biotechnology organisms. The technology uses retrons, an immune system that produces DNA, to efficiently modify genes, with varying success rates across different species.
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 identified a CRISPR variant that distinguishes tumor DNA from healthy DNA and selectively cuts the former. This method relies on methyl groups attached to DNA, which are altered in cancer cells.
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.
Researchers have created genetically modified marmosets with a knocked-out OTOF gene, replicating key characteristics of human deafness. The animals developed normally but were deaf from birth, offering a crucial tool for developing new therapies.
Researchers discovered that DNA twisting plays a significant role in CRISPR's mistakes, compromising safety and efficacy. The study used tiny DNA circles called minicircles to capture interactions between CRISPR and DNA, providing insights to help eradicate errors altogether.
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 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.
A common CRISPR platform has been established for comparative analysis of multicellularity across different species of social amoebas. This technique enables gene modifications in several Dictyostelia species, ranging from ancestral to more complex groups.
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.
Researchers developed a novel CRISPR-based technology called pPro-MobV that can remove antibiotic-resistant elements from bacterial populations. The new tool uses gene-drive thinking and has the potential to combat antibiotic resistance in healthcare settings, environmental remediation, and microbiome engineering.
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.
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
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.
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.
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.
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 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 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 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.
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.
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.
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 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.
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.