Articles tagged with Gene Editing
Researchers have shown that a single normal copy of a defective gene can prevent cysts in polycystic kidney disease. Additionally, they found that a type of drug called a glycoside can sidestep the effects of the defective gene and prevent cyst formation.
Researchers developed an Integrated Classifier Pipeline (ICP) tool to analyze CRISPR edit outcomes and track unintended 'bystander' edits. The ICP system provides a genetic fingerprint of how material is being inherited, helping scientists untangle complex biological issues.
Researchers successfully demonstrate CRISPR-Cas gene editing technology to eliminate all traces of the HIV virus from infected cells in laboratory settings. The study aims to develop a robust and safe combinatorial regimen to target diverse HIV strains across various cellular contexts.
Researchers have developed a new CRISPR-based approach to study the function of immune genes, bypassing challenges of modifying immune cells. By targeting stem cells that produce immune cells, scientists can knock out specific genes in different cell types at various times, providing greater insight into gene interactions and their rol...
Researchers created novel gene editing enzymes with improved precision, reducing off-target RNA edits by over 99%. The technology has potential applications in treating mitochondrial genetic diseases and may lead to transformative treatments within the next five years.
A Husker research duo has won a $25,000 prize in the NIH’s Targeted Genome Editor Delivery Challenge. They will advance development of universal milk exosomes capable of transporting gene editors to any location in the body, overcoming a significant challenge in using gene editing to treat disease.
Researchers developed a faster test for mpox using CRISPR and nanopore sensing technology, which can detect the virus in 32-55 minutes, compared to hours of current lab testing. The test is specific to mpox and was confirmed not to detect other viruses.
A breakthrough gene-editing therapy has been successfully treated over ten patients with hereditary angioedema, reducing symptoms by up to 95% and potentially providing a permanent cure. The therapy targets the KLKB1 gene responsible for producing plasma prekallikrein.
A new CRISPR delivery method enables precise targeting of specific cell subsets in living animals, paving the way for programmable gene therapy. The system uses antibody-targeted 'enveloped delivery vehicles' to selectively edit T-cells and create CAR T-cells.
Researchers used CRISPR to modify a tomato gene, resulting in reduced water consumption without affecting crop quality. The discovery holds implications for basic scientific knowledge and could help increase plant yields in dry conditions.
Researchers have developed CRISPR off-switches to mitigate off-target effects, a major concern in genome editing. The new technology, based on anti-CRISPR proteins, can block CRISPR-Cas3 machine function and prevent unintended edits.
A UC Riverside research project will focus on changes in the gene network that specify early intestinal precursor cells in nematodes like Pristionchus pacificus. Gene networks describe how genes turn each other on and off, and changes in these networks can lead to diseases such as cancer.
Researchers develop a delivery system for prime editing, enabling the correction of disease-causing genetic mutations in animal models. By adapting virus-like particles to carry the machinery for prime editing, scientists have achieved partial restoration of vision in mice with two genetic disorders.
A team of researchers has developed a promising gene-editing strategy for spinal muscular atrophy (SMA), a devastating pediatric neuromuscular disorder. The approach involves using CRISPR base editing to activate the SMN2 gene, which is similar to the mutated SMN1 gene responsible for SMA.
The Genes & Health study has enrolled its 10th participant in a gene-editing clinical trial for heart disease, specifically familial hypercholesterolemia. This milestone marks an important step towards improving health outcomes for people of Pakistani and Bangladeshi descent.
Scientists at Florida State University produced the first high-resolution images showing magnesium ions playing a crucial role in CRISPR-Cas9's DNA-cutting process. The discovery sheds light on how magnesium coordinates double-stranded breaks, providing new insights into the enzyme's functioning.
Researchers improved biomass-related traits in sheepgrass using a custom genome editing system, revealing its potential for rapid genomic breeding. The study increased understanding of sheepgrass genomics and established a precedent for its genetic improvement.
Researchers employed heat-induced FT overexpression to accelerate assessment of floral phenotypes after CRISPR knockout of LEAFY and AGAMOUS genes in poplar trees. The study successfully induced early flowering and demonstrated a wide range of inflorescence and floral forms.
Researchers have identified a new mechanism by which phages evade CRISPR-Cas immune systems in bacteria, revealing a potential approach to make gene editing safer and more efficient. This discovery could lead to the development of bespoke anti-CRISPRs to neutralize CRISPR-Cas systems and provide an alternative to antibiotics.
A new CRISPR-based gene-editing tool, AsCas12f, has been developed with enhanced editing ability and compact size. The engineered enzyme has already shown success in animal trials and holds promise for improved treatments of genetic disorders.
Researchers create adenine base editor with 'on/off' switch, reducing off-target edits by over 70% and increasing accuracy of on-target edits. The tool has potential to correct nearly half of disease-causing point mutations in human genome.
A CRISPR-Cas3 system has restored dystrophin protein function in induced pluripotent stem cells from patients with Duchenne muscular dystrophy. The approach uses a dual CRISPR RNA method to remove large sections of the dystrophin gene, yielding truncated but still functional proteins for various mutation patterns.
Researchers used base editors to introduce specific combinations of activating and inactivating mutations into healthy organoids, creating realistic models for various types of cancer. This allows for further investigation into the development and treatment of cancer, with potential applications including testing new drugs.
A novel CRISPR-based gene-editing treatment, EBT-001, effectively removes SIV from the genomes of non-human primates without off-target effects. The study's findings support the development of a cure for HIV/AIDS in humans and pave the way for ongoing clinical trials.
City of Hope will conduct Phase 1 clinical trials for novel cell and gene therapy treatments for patients with HIV, acute myeloid leukemia, and severe aplastic anemia. A CAR T therapy for HIV is also being developed to potentially target other diseases.
Researchers at the Marine Biological Laboratory have devised a method to precisely alter rotifer genomes using CRISPR-Cas9, enabling the study of fundamental biology and evolution. The new approach will allow scientists to investigate various aspects of biology, including aging, DNA repair mechanisms, and mitochondrial function.
Researchers at the University of Arkansas successfully used gene editing to reduce 'chalkiness' in rice, a condition caused by heat stress. The modified rice lines showed a significant improvement in grain quality, with increased starch granule compactness and reduced chalkiness.
CHOP and Penn Medicine researchers have developed a proof-of-concept model for delivering gene editing tools directly into diseased blood cells within the body. This approach aims to reduce costs and increase access to gene therapies for blood disorders, which currently require chemotherapy and stem cell transplants.
Researchers at Cornell University have discovered a mutation in the MdLAZY1A gene responsible for the 'weeping' growth pattern in apple trees. This finding could lead to more productive and labor-saving orchards by allowing branches to grow downwards, thereby increasing resource allocation towards reproductive growth.
Gang Bao's lab receives a 4-year, $2.6 million grant from the National Institutes of Health to investigate the safety and efficacy of using gene editing treatments like CRISPR-Cas9 to treat sickle cell disease. The team aims to understand the mechanisms behind large gene modifications and their biological consequences.
Researchers at North Carolina State University used CRISPR gene-editing to breed poplar trees with reduced lignin levels, while improving their wood properties. This breakthrough could make fiber production for paper, diapers, and other products more sustainable, efficient, and cost-effective.
Hematopoietic stem cell culture technology improves genome editing in HSCs by increasing successful correction rates to 100%, eliminating genetic mutations, and enhancing cell transplantation outcomes. This breakthrough enhances the efficiency and safety of gene editing in treating genetic diseases.
A new study by researchers at NYU and the New York Genome Center combines deep learning with CRISPR screens to control human gene expression. The model predicts on- and off-target activity of RNA-targeting CRISPRs, enabling precise gene controls for developing new therapies.
A new study from Aarhus University has found that applying AI predictions of protein structures enhances the CRISPR technology, making the cuts in a patient's DNA more precise. This discovery may lead to better treatments for patients with genetic disorders and potentially develop cures for various genetic diseases.
Duke researchers discovered a new approach to CRISPR RNA variants that can specifically target challenging areas of DNA for editing. This improvement enables the repair of mutations associated with more diseases, leading to safer and more effective gene editing therapy.
A new study reveals a consistent difference in favorability ratings between gene editing and genetically modified organisms (GMOs) in social and traditional media. Gene editing consistently receives higher favorability ratings, with close to 100% achieved in numerous monthly values, indicating a positive shift in public sentiment.
Researchers have uncovered a new RNA-guided DNA-cutting enzyme called Fanzor in eukaryotes, which has the potential to edit the human genome with precision. The system was found to be more easily delivered to cells and tissues than CRISPR/Cas systems, making it a valuable new technology for human genome editing.
Researchers have discovered that gene editing technologies may introduce unintended mutations and damage to DNA in early human embryos. The study found that most cells repair breaks in the DNA using non-homologous end joining, which can lead to additional genetic abnormalities.
A new gene editing tool exploits a bacterial immune system to target and eliminate antibiotic-resistant genes. The tool has shown early promise in laboratory experiments by protecting host cells from developing resistance and reversing it in resistant hosts.
Scientists at Temple University have developed a novel gene-editing strategy that disrupts the ability of HIV-1 virus to enter host cells by targeting a rare genetic disorder. This approach may offer another target for developing next-generation CRISPR technology for HIV elimination, while avoiding adverse effects on cell mortality.
Researchers have developed chicken eggs without the protein that causes egg white allergies, confirming their safety profile. The modified eggs, called OVM-knockout, were tested and found to be free from ovomucoid proteins, making them a potential solution for individuals with egg allergies.
Researchers at KAIST have developed a new sRNA tool that can effectively inhibit target genes in various bacteria, including both Gram-negative and Gram-positive bacteria. The BHR-sRNA system was shown to suppress pathogenicity in antibiotic-resistant pathogens and improve industrial strains for high-value-added chemical production.
Researchers have engineered a new CRISPR-based drug candidate targeting E. coli directly while preserving the microbiome. The innovative treatment has shown promise in reducing E. coli burden in mice and is now in phase 1 clinical trials to treat blood cancer patients and prevent deadly infections.
Scientists have developed a new method to deliver genetic information to stem cells using nanoparticles coated with a specific polymer, enabling more efficient control over cellular differentiation. This innovation has the potential to improve the efficiency and effectiveness of regenerative medicine treatments.
A new approach, STING-seq, combines genetic association studies, gene editing, and single-cell sequencing to identify causal variants and genetic mechanisms for blood cell traits. This method can help scientists identify drug targets for diseases with a genetic basis.
Researchers at Temple University and the University of Nebraska Medical Center developed a novel dual gene-editing approach that can effectively eliminate HIV infection. The therapy targets both HIV-1, the virus responsible for AIDS, and CCR5, a co-receptor that facilitates viral entry into cells.
The MBL's 36th year Logan Science Journalism Program provides immersive research training for twelve fellows from prominent news organizations. They will participate in Biomedical and Environmental Hands-On Research Courses, exploring cutting-edge technologies and scientific innovations.
Researchers developed a new approach to genetic engineering of cells, promising improvements in speed and efficiency over current methods. The technique uses special cell-penetrating peptides to deliver CRISPR-Cas molecules into cells with up to 100% efficiency and low toxicity.
Researchers at Gladstone Institutes have made a groundbreaking discovery about how neurons consume and metabolize glucose, a process crucial for maintaining normal energy levels. The study found that neurons rely on glycolysis to break down glucose, and its disruption can lead to severe learning and memory problems in mice.
Researchers developed an optimized genome-editing method that vastly reduces mutations, enabling more effective treatment of genetic diseases. The new technique uses a 'safeguard gRNA' to control DNA cleavage, reducing off-target effects and cytotoxicity.
Researchers at the University of Tokyo have discovered the 3D structure of TnpB, a protein involved in genome editing and a probable precursor to the CRISPR-Cas12 enzyme. The study reveals how TnpB recognizes and cuts DNA using a unique pseudoknot shape similar to that found in guide RNAs of Cas12 enzymes.
Researchers have developed a new method for downregulating gene translation in plants using upstream open reading frames (uORFs). The study, published in Nature Biotechnology, demonstrates the potential for precise and incremental regulation of gene expression.
A new study led by OHSU researchers reveals that gene editing technology in human embryos can lead to unintended changes in the genome and may not accurately reflect gene edits. The study highlights the need for caution when using genetically edited embryos to establish pregnancies.
Researchers developed a machine learning algorithm to predict the chances of successful prime editing gene edits, assessing thousands of DNA sequences and identifying key factors such as sequence length and DNA repair mechanisms. The tool promises to speed up efforts to bring prime editing into the clinic.
Researchers from Rice University have developed a new approach to control gene expression using proteolysis targeting chimeras (PROTACs). By reengineering the PROTAC molecular infrastructure, they demonstrated the ability to achieve chemically induced dimerization (CID), allowing for precise control over gene activation in specific loc...
Researchers at UW-Madison developed silica nanocapsules that can carry CRISPR tools across the blood-brain barrier, enabling brain-wide gene editing for disorders like Alzheimer's and Parkinson's. The technology has potential for non-invasive delivery of gene therapies.
The CABBI team successfully demonstrated precision gene editing in miscanthus, a promising perennial crop for sustainable bioenergy production. The results will accelerate efforts to tap the huge potential of this highly productive but genetically complex grass as a source for biofuels, renewable bioproducts, and carbon sequestration.
Researchers developed a flexible genetic hacking system to convert split gene drives into full gene drives, enabling safe testing and potential real-world applications. The new system revealed surprising fitness costs of full drive systems, with slower-than-predicted spread rates in cage experiments.
A new breeding strategy enables rapid production of tomatoes with various fruit colors, including red, yellow, pink, and green, using CRISPR/Cas9-mediated multiplex gene editing. This method requires less time and produces transgene-free plants with desirable traits, offering a promising approach for improving multigene-controlled traits.
A new approach to gene therapy for inherited blindness uses lipid nanoparticles to deliver mRNA inside the eye, targeting light-sensitive cells and creating proteins that edit vision-harming gene mutations. The technology has shown promising results in animal studies, including mice and nonhuman primates.