Articles tagged with Genome Editing
A team of researchers at Harvard's Wyss Institute and ETH Zurich have developed a computational approach to identify genomic safe harbors (GSHs) with high potential for safe insertion of therapeutic genes. The study validated two GSH sites in adoptive T cell therapies and in vivo gene therapies for skin diseases.
Gladstone Institutes researchers have pioneered a new method to edit genes in human cells using retrons, which can produce abundant copies of template DNA from inside cells. The optimized system has shown improved efficiency and precision compared to current approaches.
Researchers have successfully genome-edited vascular endothelial cells using a novel nanoparticle delivery system, which could treat diseases such as acute respiratory distress syndrome and coronary artery disease. The technology has the potential to correct gene mutations, promote vascular repair, and inhibit vascular injury.
Scientists have developed a new method using CRISPR-Cas9 to target specific fat cells, reducing the time and cost of genetic discovery in obesity research. The technique allows researchers to study genes in brown adipose tissue, which plays a crucial role in regulating body temperature.
Researchers have developed Find Cut-and-Transfer (FiCAT) technology, a tool capable of accurately writing small and large genes. FiCAT allows precise insertion of large fragments into the genome, enabling development of therapeutic solutions for diseases like Duchenne muscular dystrophy and hereditary blindness.
Researchers at UC Berkeley have developed a new CRISPR editing technology that enables simultaneous editing of genes in multiple cell types and species within a microbial community. This approach, called community editing, has the potential to track edited microbes and understand their functions within complex ecosystems.
Researchers have expanded the number of naturally occurring CRISPR-Cas systems, giving a wealth of potential new tools for large-scale gene editing. The discovery could lead to treating complex diseases associated with multiple genes.
Researchers at Okayama University used CRISPR/Cas9 technology to genetically edit barley, creating mutants that resist pre-harvest sprouting and can be promoted with cold temperatures or hydrogen peroxide treatment. This breakthrough has significant implications for high-quality barley production and could lead to improved beer quality.
Researchers at McGill University developed a new technique to manipulate DNA methylation levels at specific genes using CRISPR/Cas9 technology. This approach enables targeted demethylation of genes associated with diseases, such as insulin gene dysregulation in diabetes.
Researchers at Princeton University developed a novel method called Repair-seq to understand genome editing tools, revealing complex mechanisms of DNA repair. This work improves the CRISPR gene-editing method by identifying new pathways and optimizing systems.
The Science Communication Lab has received a 3-year NSF grant to create new films and virtual workshops about novel breakthroughs and techniques in science. The lab aims to expand its work across filmmaking and education to reach broad and diverse audiences, emphasizing diversity, equity, and inclusion.
Researchers have established an organoid biobank to search for genes essential for SARS-CoV-2 replication and spread. The study identified TMPRSS2 as a potential therapeutic target for the coronavirus, with specific inhibitors recently developed.
The study contributes to deciphering genetic background of unique marsupial characteristics by creating genetically modified opossums. The researchers successfully edited a gene responsible for body pigments, producing albino offspring with inherited genes.
A new genetic engineering platform has been established in methylotrophic yeast Pichia pastoris, enhancing homologous recombination rates and genome editing efficiency. This breakthrough can enable the stable loading of over 100 exogenous genes and precise regulating of gene expression.
Ben Ouagrham-Gormley is researching ethical issues related to human germline genome editing, a key aspect of China's global scientific strategy. The researcher will analyze data and conduct interviews with Chinese and U.S. scientists and government officials.
A new CRISPR/Cas9 variant, miCas9, has been developed to improve the efficiency of gene editing while reducing unintended insertions or deletions. This advancement holds promise for advancing gene editing in genetic research and potentially treating diseases.
The National Human Genome Research Institute has published its 2020 Strategic Vision, which describes cutting-edge research priorities and opportunities in human genomics. The vision identifies four focus areas: guiding principles, sustaining and improving genomic research, breaking down barriers, and compelling biomedicine projects.
A joint EU conference explores GMOs, emphasizing EU regulation prospects and optimal use of scientific findings for consumer protection. The event features a series of free online webinars with prior registration.
A global citizens' assembly consisting of at least 100 lay citizens from around the world would explore and deliberate on genome editing technologies and regulatory principles. This assembly aims to address the lack of consistent governance and bridge gaps between community values, improving overall governance of genome editing technology
Researchers propose national and global citizens' assemblies to examine the ethical and moral issues surrounding genome editing, involving laypeople from diverse backgrounds. The goal is to develop moral and political regulation and ensure fair access to the technology.
Researchers developed polymeric carriers that can load multiple types of genetic material, improving efficiency and reducing toxicity compared to existing analogues. The technology has the potential to revolutionize gene therapy by delivering two RNA and DNA molecules through a single carrier.
Researchers have identified a potential new treatment for dilated cardiomyopathy (DCM) using an acne drug. A single mutation in the RBM20 gene was found to cause the disease, and increasing expression of this protein may overcome the insufficient expression seen in patients with DCM.
Scientists from the University of Tsukuba found that retrotransposons may change how genes are expressed in melons. In a study published in Communications Biology, researchers discovered that retrotransposons had a role in altering gene expression when melon genomes were diversifying and affecting fruit ripening.
The EU is taking a cautious approach to gene drive organisms, considering their potential risks and benefits. The technology uses CRISPR/Cas-9 genome editing to introduce permanent changes in ecosystems, raising concerns about long-term ecological impact.
A new method has been developed to analyze pathogenic DNA repeats in the human genome, revealing their role in genetic diseases such as Fragile X syndrome. The technique combines nanopore sequencing, stem cell culture, and CRISPR-Cas technologies to provide detailed insights into these previously inaccessible regions.
Researchers at the University of California, Davis have successfully genome-edited a dairy bull to prevent it from growing horns, and their findings show that none of its offspring developed horns. The study also highlights the need for screening and selection to address plasmid integration when using genome-editing in livestock.
The Carnegie Mellon and Yale research team will scale up production of PNAs, improve DNA binding properties, and develop new nanoparticle formulations for enhanced in vivo editing. The goal is to move the technique closer to clinical therapeutic applications.
Researchers at UW-Madison have developed tiny customizable nanocapsules to deliver gene-editing payloads, reducing the risk of unintended genetic edits. The nanocapsules are expected to improve the efficiency and safety of gene therapies, potentially treating inherited diseases and stubborn viral infections.
A multistakeholder study proposes six policy options for governing heritable genome editing, including proactive regulation and broad public engagement. Meanwhile, gynecological surgery reform is urged due to declining OB-GYN skills and lack of transparency, putting patient safety at risk.
Genome editing enables crop development with resistance to pests, diseases, drought, and heat, reducing losses and chemical pesticide sprays. However, regulatory hurdles in Europe may hinder the adoption of these technologies, posing a risk to developing countries.
Researchers at UC Davis have made a breakthrough in plant biology by discovering a way to produce crops as clones from seed. This process, known as apomixis, allows for the reproduction of high-yielding and disease-resistant crops without the need for expensive hybrid seeds. The discovery has significant implications for global food se...
Researchers at Baylor College of Medicine have successfully used genome editing to correct a genetic mutation causing a rare and deadly heart condition. The study uses CRISPR/Cas9 technology to selectively disrupt the disease-causing gene, reducing arrhythmia symptoms in mice. This breakthrough could lead to a permanent treatment for p...
The NIH has awarded a $3.6 million grant to study the safety of CRISPR-like therapies in human tissues. A team led by Todd McDevitt will assess the safety and toxicity issues of genome editing, developing platforms to detect adverse effects on physiological function.
Researchers have developed a new crop through genome editing, combining the benefits of wild plants with desirable traits. The new tomato variant boasts increased antioxidant content, larger fruit size, and improved compact growth, offering potential health benefits.
Researchers aim to elucidate biological function of DPLG1-4 genes, which are co-opted by organisms to build new gene regulatory pathways linked to reproduction. They will use CRISPR-Cas9 genome editing techniques on fruit flies to test hypotheses.
A study by University of Oulu researchers has identified novel genes and mechanisms associated with aggressive prostate cancer progression. The research suggests ways to improve risk stratification and clinical treatment for advanced prostate cancer.
Researchers at Penn School of Medicine successfully reduced cholesterol levels in rhesus macaques using genome editing, offering a new approach to treating heart disease patients who don't tolerate PCSK9 inhibitors. The study demonstrates the potential of this therapy for large-scale clinical trials and future treatment options.
A new rabbit model with clinical similarity to human patients has been developed for Duchenne muscular dystrophy (DMD) research. The model exhibits signs of muscular dystrophy, including impaired physical activity and muscle loss, making it a promising tool for advancing DMD research and developing novel therapies.
Researchers are investigating genome elimination in organisms, a process crucial for survival and evolution. Studies aim to uncover the mechanisms of this complex phenomenon, which could lead to the treatment of hereditary diseases.
The review discusses potential applications of genetic modification and genome editing in cattle for food production, highlighting the need for clear information about methods and their consequences. The authors emphasize the importance of stakeholder involvement and international cooperation to address ethical concerns and ensure publ...
The new eMAGE method eliminates DNA breakage errors, enabling efficient genetic modifications with high accuracy. This technology has the potential to revolutionize disease treatment, antibiotic production, and industrial biotechnology applications.
A survey of genetics professionals found high support for research into somatic uses of gene editing, but more divided views on germline uses. Most geneticists felt it would be acceptable for therapeutic purposes in the future, differing from public opinions.
Researchers at Max Planck Florida Institute for Neuroscience have developed a new tool called vSLENDR, which enables precise genome editing in post-mitotic neurons of the brain. This breakthrough technology utilizes CRISPR-Cas9 and an adeno-associated virus to increase the efficiency and flexibility of HDR DNA repair.
The Leopoldina Annual Assembly explores the ethical, legal, and technological aspects of genome editing. Researchers will discuss its applications in humans, plants, and animals, as well as its potential impact on food production and human therapies.
Researchers used CRISPR-Cas9 genome editing to study the optix gene's role in butterfly wing patterns. In four species, deleting optix resulted in black pigment replacing red and orange pigment, with changes in expression of genes involved in pigment production.
A group of 11 organizations has issued a statement on germline genome editing in humans, recommending against human pregnancy-related editing and supporting publicly funded in vitro research. The statement outlines scientific and societal steps necessary before implementation of such clinical applications is considered.
The National Institutes of Health has instituted a policy to consider sex as a biological variable in funded research, with recommendations provided by coauthors Terri Lynn Cornelison and Janine Austin Clayton. Guidelines include including both male and female subjects in studies and generalizing findings appropriately.
Dr. Tuuli Lappalainen is awarded a 5-year, $1.7 million NIH grant to investigate why identical genetic mutations cause disease in some individuals but not others. The study aims to understand the role of haplotype epistasis in human evolution and disease.
Researchers find that members of the DUX family of proteins, specifically DUX4, trigger gene expression program in human embryos. DUX4 stimulates expression of genes induced during zygotic genome activation by binding to their regulatory regions.
The Jackson Laboratory is developing a high-throughput approach to improve the efficiency of targeted nuclease-mediated HDR for genome editing. The goal is to significantly enhance the reliability and accuracy of CRISPR-Cas9 technology, enabling faster and more cost-effective therapeutic delivery.
Genome editing offers promise for treating genetic disorders but raises major technological and ethical concerns. The ACMG Board of Directors emphasizes the need to overcome current limitations and address issues such as off-target effects and epigenetic marks.
Researchers have successfully corrected the mutated gene responsible for sickle cell disease in stem cells using CRISPR-Cas9 gene editing. The study holds promise for a new treatment by re-infusing edited stem cells into patients, potentially alleviating symptoms and improving lifespan.
Recent advances in genomics, physiology, and genetic manipulation enable the bioengineering of new strains of algae. Eukaryotic microalgae are being utilized to produce environmentally friendly, renewable biomaterials and biofuels.
Researchers at Cornell University and Florida State University identified a tiny percentage of regulatory DNA in the maize genome that accounts for roughly half of the variation in observable traits found in corn. This discovery enables breeders to focus on these areas for more efficient plant breeding.
Researchers identified a novel syndrome resulting from multiple genomic lesions in two male siblings with similar phenotypic features. Whole exome sequencing and cytogenetic testing revealed terminal duplications of Chromosome 16q and deletions of Chromosome 5p, which likely contribute to the complex clinical presentation.
A new research essay in Cell journal suggests that the US can follow the UK's approach to regulate germline editing and mitochondrial replacement therapy. The authors argue that the UK's experience with MR therapy provides a well-drawn regulatory framework for evaluating the safety and ethics of germline editing.
McGill University researchers suggest a model for guiding policy on germline gene editing, based on pre-implantation genetic diagnosis after in vitro fertilization. The PGD model provides a robust approach to regulation, outlining medically determined requirements and substantial risk of occurrence for genetic testing.
Researchers at Broad Institute of MIT and Harvard have engineered changes to the CRISPR-Cas9 system, significantly cutting down on 'off-target' editing errors. The newly-engineered enzyme, eSpCas9, will be useful for genome editing applications requiring high specificity.
Researchers presented various applications of Bio-Rad's Droplet Digital PCR, including copy number determination, genome editing, and liquid biopsy. The technology demonstrated precision, reproducibility, and sensitivity in detecting complex genomic rearrangements and biomarker levels.
The National Academies have launched an international initiative to address the technical, social, and ethical issues surrounding human genome editing. The initiative aims to provide a comprehensive understanding of human genome editing and its implications for improving human health and boosting food production.