Articles tagged with Gene Editing
Researchers have used CRISPR gene editing to study the regulation of the Unusual Floral Organs (UFO) gene in plants, uncovering the importance of conserved non-coding DNA sequences in controlling flower formation.
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 from Flinders University applied gene editing to explore the role of enzyme ACE2 in healthy placental development. They found that ACE2 plays a key role in helping cells grow properly and that a genetic variation is linked to major pregnancy complications.
Researchers successfully developed CoQ10-producing rice through targeted gene editing, offering a cost-effective approach to nutritional fortification. The discovery provides great potential benefit for human health, particularly heart protection, and expands the food sources of CoQ10.
Researchers at UCSF used CRISPR gene editing technology to transform ordinary white fat cells into 'beige' fat cells that voraciously consume calories to make heat. Implanted near tumors, these cells outcompeted cancer cells for nutrients, beating back five types of cancer in lab experiments.
Scientists create complex human cell lines with random genome structural changes to study their impact on cell survival and gene expression. They found that essential genes must remain intact for significant structural changes to be tolerated.
UCSF researchers found that when brain cells of female mice express only a maternal X chromosome, their memory and cognitive skills deteriorate faster. This study could explain the variation in brain aging between the sexes and individual women.
A recent study from the University of Illinois shows that gene-edited bacteria can supply equivalent of 35 pounds of nitrogen from air during early corn growth, increasing vegetative growth, nitrogen accumulation and yield by an average of 2 bushels per acre.
Researchers have developed a method for delivering therapeutics to targeted locations in the human body using universal milk exosomes. This technology could be used to treat common and rare diseases alike, offering a potential boon to rare disease communities.
Researchers developed a gene-editing treatment that reduces prion protein levels in the brain by up to 60% and extends mouse lifespan by about 50%. The base-editing approach could lead to a one-time treatment for prion disease patients.
Lehigh University bioengineering researcher Tomas Gonzalez-Fernandez is exploring how combining CRISPR with biomaterials can improve gene editing's safety and efficacy for therapeutic use. His NSF CAREER award-funded research aims to develop more targeted and controlled therapies for genetic diseases.
Researchers have developed a new genetic engineering tool, mvGPT, that can precisely edit genes, activate gene expression, and repress genes all at the same time. The technology has shown promise in treating genetic diseases such as Wilson's disease and type I diabetes by targeting multiple genetic conditions simultaneously.
Researchers developed a gene therapy approach to treat chronic hypereosinophilia by delivering an anti-human eosinophil antibody via AAV-based gene therapy. The therapy successfully suppressed blood eosinophil levels in mice, showing promise as a potential treatment for the condition.
A new gene editing tool called SPLICER has been applied to reduce the formation of amyloid-beta plaque precursors in a mouse model of Alzheimer's disease. The application shows improved efficiency over current standard gene editing technology and potential for application in other diseases.
Researchers compared urine NAA levels in patients with mild and typical Canavan disease, finding lower levels in those with the milder form. This discovery has potential for a rapid and cost-effective way to screen for CD incidence and severity.
A USC Stem Cell study has identified key gene regulators that enable some deafened animals, including fish and lizards, to naturally regenerate their hearing. The researchers found a class of DNA control elements known as 'enhancers' that amplify the production of a protein called ATOH1, which induces sensory cells in the inner ear.
Branden Baptiste, 20, becomes the world's first patient to receive base editing gene therapy for sickle cell disease. The treatment has left him feeling 'more than fine' with no symptoms of the disease. After undergoing chemotherapy and a series of tests, Branden was infused with genetically treated cells in December 2023, allowing his...
A new study found that gene therapy delandistrogene moxeparvovec significantly extended the median survival of Duchenne muscular dystrophy (DMD) rats to >25 months. Additionally, the treatment elicited statistically significant improvements in cardiac parameters and mobility.
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.
Engineers at USC Viterbi School of Engineering have developed a new CRISPR toolkit that allows for precise, remote-controlled genome editing using focused ultrasound. This breakthrough enables the treatment of various genetic disorders and diseases by activating or silencing specific genes with precision.
Researchers at UCSF are enrolling patients in a clinical trial to correct the genetic mutation causing sickle cell disease using non-viral CRISPR-Cas9 gene editing. The therapy aims to eliminate the need for a bone marrow transplant and create a new blood system free of the disease.
A new gene drive technology, known as e-Drive, has been developed to reverse insecticide resistance in pests by replacing mutant genes with native ones. The system is designed to spread and then disappear, leaving only a population of insects susceptible to pesticides.
Researchers developed a novel lentivirus-based gene therapy strategy in CD34+ hematopoietic progenitor cells, which showed therapeutic levels of expression of the anti-sickling beta globin protein. Cyclosporin improved transduction efficiency and preserved cell viability.
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.
The CRISPR tool was successfully used to correct a genetic defect in cells affected by chronic granulomatous disease. However, the repair process also introduced new genetic defects, highlighting the need for caution when using CRISPR technology in clinical settings.
Using state-of-the-art technology, researchers identified specific steps needed for CRISPR to become active and perform its gene editing function. Understanding these mechanisms could lead to improved designs for CRISPR-based gene editing.
Researchers used ex vivo lentiviral gene therapy to treat MPS IVA in mice, achieving partial correction of bone pathology and complete correction of heart pathology. The study suggests potential for novel therapies to treat patients with MPS IVA.
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 USC Stem Cell mouse study identifies a small subset of blood stem cells as the primary driver of immune aging. The researchers found that this subset overproduces innate immune cells, leading to an age-associated imbalance and increased disease risk. By targeting this subset, the study suggests a potential therapy to delay immune agi...
Dr. Fyodor Urnav proposes a set of initiatives to address the crisis, including pooling patients by syndrome and permitting multiple gene editors in a single Investigational New Drug application. This approach aims to accelerate the development of CRISPR therapies for rare genetic diseases.
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.
Researchers at Lehigh University are developing predictive models for gene editing with CRISPR to improve outcomes and expand medical applications. The team is using AI and advanced computer models to simulate the effects of altering a single gene on the entire genome, enabling them to predict and avoid unintended consequences.
A research team developed an RNA-based sensor platform that can regulate gene expression in bacteria, mimicking natural biological interactions. The START platform enables tunable control over sensor response and detection of various molecules, including drugs and proteins.
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%.
Ling Li's groundbreaking research improves global nutrition and sustainability by increasing protein content in rice and soybean crops. The gene-edited approach boosts nutritional value while promoting sustainable agricultural practices.
CAR-T cell therapy, a regenerative immunotherapy, has shown promise in treating blood cancers but struggles with T-cell exhaustion. Researchers have discovered that overproduction of the IL-4 protein causes this exhaustion and used CRISPR gene-editing technology to remove it, improving CAR-T cell therapy outcomes.
Researchers have shown that repeated administration of lipid nanoparticle-encapsulated mRNA therapy significantly extended survival and reduced serum leucine levels in a mouse model of maple syrup urine disease. The treatment approach may represent a potential long-term universal treatment for MSUD.
Researchers at the University of Hawaii have developed a new gene editing technology that can efficiently deliver healthy genes to the body. This method addresses limitations of current methods and has shown success rates of up to 96%, potentially leading to faster and more affordable treatments for various genetic diseases.
Researchers are exploring the use of CRISPR Prime Editing to optimize CAR-T cells for treating multiple myeloma and other cancers. The goal is to improve effectiveness, duration, and cost-effectiveness of CAR-T cell therapies.
Researchers used gene editing to restore hearing in adult mice with a form of inherited deafness called autosomal dominant deafness-50 (DFNA50). The approach involved shutting down a damaged copy of the microRNA (miRNA) gene, which enabled the animals to regain hearing. This method may eventually lead to potential treatments for inheri...
Researchers at Broad Institute of MIT and Harvard have developed a gene-editing approach that efficiently corrects the most common mutation causing cystic fibrosis, found in 85% of patients. The new method precisely and durably corrects the mutation in human lung cells, restoring cell function to levels similar to Trikafta.
Researchers developed a CRISPR/Cas9 genome editing approach to target and disrupt a specific mutation causing progressive hearing loss. The study found robust preservation of auditory function, with earlier intervention proving most optimal.
A team of scientists at Gladstone Institutes has developed a new method that enables them to make precise edits in multiple locations within a cell—all at once. They created a tool using molecules called retrons to efficiently modify DNA in bacteria, yeast, and human cells.
Researchers, including Assistant Professor James Lewis, are studying the evolution of butterfly wing color patterns as a model for understanding population adaptation to environmental changes. The study aims to understand why some Heliconiine butterflies lose their mimicry phenotype and how this affects their survival.
Researchers at the University of Sydney have developed SeekRNA, a programmable tool that can precisely target and relocate genetic sequences with high accuracy and flexibility. This breakthrough technology surpasses current limitations of CRISPR, enabling more precise editing and reducing errors.
Researchers developed an ex vivo gene-editing protocol to treat hereditary tyrosinemia type 1 using liver cell therapy. The study showed robust engraftment and expansion of transplanted gene-edited hepatocytes in recipient mice, correcting disease indicators such as normalized tyrosine, phenylalanine levels.
New research reveals that CRISPR/Cas9 gene editing tools have biases against cells from people of African ancestry, leading to false negative results. The study's findings highlight the importance of increasing genetic diversity in large-scale cell line libraries to mitigate this bias.
Scientists have successfully edited genes in a highly resilient tardigrade species using CRISPR technology, allowing them to investigate the genetic basis of its exceptional abilities. The research could lead to new insights into human organ preservation and transplantation.
Scientists have improved a gene-editing technology that can insert or substitute entire genes in the genome, potentially treating multiple genetic diseases with a single therapy. The new approach, eePASSIGE, uses prime editors and recombinase enzymes to make gene-sized edits several times more efficiently than previous methods.
Researchers found that Orco protein is necessary for the development and life of olfactory cells in ants. Mutant ants lacking Orco experience massive olfactory neuron loss, suggesting a link between Orco and neuronal survival.
Researchers have developed a compact, high-fidelity version of the Cas12a protein, which can be packaged within a non-pathogenic virus for targeted gene editing. The modified protein demonstrates efficient editing activity and has been shown to reduce blood cholesterol levels in mice with high cholesterol.
Researchers at Rice University have identified genes critical for enteric nervous system development using zebrafish embryos. Opioid signaling pathways are required for the formation of nerves in the gut, contradicting previous assumptions about their role in pain perception and addiction.
Researchers have developed a new gene-editing system called multiplexed orthogonal base editors (MOBEs) to tackle complex diseases. MOBEs enable simultaneous installation of multiple point mutations across the genome, reducing the risks associated with traditional gene-editing tools like CRISPR-Cas9.
A KAUST team developed a simple approach to tackle CRISPR's deletion issue by targeting error-prone DNA repair pathways. By modulating specific genes, they reduced large deletions while enhancing homology-directed repair efficiency.
Researchers at Fred Hutch Cancer Center have made progress in laboratory studies using gene editing to eliminate herpes virus infections. The experimental therapy eliminated 90% of HSV-1 after facial infection and 97% after genital infection, reducing viral shedding.
A pioneering CRISPR gene editing trial has demonstrated significant improvement in vision for 79% of participants with inherited retinal degeneration. The study's findings support further research into the potential of CRISPR-based treatments for inherited blindness.
A phase 1/2 trial of CRISPR gene editing has demonstrated safety and efficacy, with measurable improvements in 11 out of 14 participants with a form of inherited blindness. The treatment, EDIT-101, was found to be clinically meaningful for four participants and showed significant improvements in cone-mediated vision.
Researchers identified a small RNA-binding protein called La that promotes gene editing with high efficiency. The team created a new protein, PE7, which harnesses La's activity to enhance prime editing, leaving unwanted byproducts at low frequencies.
Researchers used CRISPR-Cas9 to genetically modify kissing bugs, opening a door to controlling Chagas disease. The new method, ReMOT Control, allows efficient and targeted editing of genes in eggs.