Articles tagged with Viral Gene Delivery
Researchers developed a new approach using the microbial protein Archaerhodopsin-3 to induce apoptosis in cancer cells, leading to significant tumor shrinkage when exposed to green light. The findings, published by Okayama University, show great potential for this light-activated molecule as a novel cancer therapy.
A team of scientists from SR-Tiget has identified a unique window shortly after birth to deliver lentiviral vectors directly into the bloodstream, enabling gene transfer and long-term engraftment. This approach shows promise for treating some genetic blood disorders without stem cell transplantation or chemotherapy.
A new gene therapy has successfully restored immune function in nine children with severe leukocyte adhesion deficiency-I (LAD-I), a rare genetic disorder. The treatment has eliminated symptoms and reduced severe infections, offering a life-changing benefit to these patients.
Researchers at MIT developed a control circuit that can precisely regulate gene expression levels, improving the efficacy and safety of gene therapy treatments. The 'COMMAND' circuit uses microRNA to suppress gene expression, allowing for tighter control over treatment outcomes.
ENVLPE addresses limitations of previous gene editing delivery systems by hijacking intracellular transport mechanisms to ensure efficient packaging and protection of gene editors. The system was tested in a mouse model of inherited blindness, achieving astounding restoration of vision, and has potential for cancer therapy advancements
Researchers have successfully reprogrammed part of the large intestine to function like the nutrient-absorbing small intestine, reversing malnutrition in a preclinical study. The technique, which deletes the colon gene SATB2, restored nutrient absorption and improved survival rates in mice with short bowel syndrome.
A multidisciplinary team has generated an atlas to optimize gene therapy delivery, providing researchers with insights into the most effective viral vectors for specific tissues. The study identifies AAV4 as a promising vector for vascular and pancreatic applications, offering new possibilities for treating conditions like diabetes.
Researchers are conducting a first-in-human clinical trial to test a modified herpes virus that targets spinal cord nerve cells to treat neurogenic bladder. The therapy, EG110A, aims to block sensory nerve signals causing involuntary bladder contractions and incontinence.
A study published in Human Gene Therapy found that over half of individuals with Niemann-Pick disease type C1 lacked neutralizing antibodies against AAV2 and AAV9. This absence of antibodies may impact the effectiveness of gene therapy treatments for this rare disorder.
Researchers have discovered a novel gene therapy that can reverse conduction slowing and prevent cardiac arrhythmias by introducing the SCN10a-short gene into heart muscle cells. The treatment has shown promise in animal models and human cell studies, offering a potential solution for millions affected by arrhythmias worldwide.
A synthetic retinoic acid-inducible gene I (RIG-I) agonist RNA has been shown to induce innate immune signaling and death of hepatocellular carcinoma cells in vitro. The addition of recombinant interferon-b potentiated this cell death, suggesting a potential new mechanism for treating patients with liver cancer.
Researchers identified a method to enhance CAR-T cell therapy by modifying the CUL5 gene. This approach improves T cells' growth and longevity, making them more effective in fighting cancer. The study suggests a new way to create targeted cells using a virus to deliver genetic material.
Researchers have developed a new gene therapy that targets aggressive brain cancer, glioblastoma, with a precise delivery system. The treatment uses a novel virus to deliver a targeting drug to cancer cells, achieving cure rates of up to 90% in mouse models.
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.
Researchers developed AI-driven therapeutic platform mimicking viral structures to deliver therapeutic genes to target cells. The innovative approach achieved precise symmetrical structures and effectively delivered payloads, paving the way for breakthroughs in gene therapies and next-generation vaccines.
A new study shows targeted delivery of energy-disrupting gene therapy using nanoparticles shrinks glioblastoma brain tumors and aggressive breast cancer tumors in mice. The technology, mLumiOpto, induces light-activated electrical currents inside cells to disrupt mitochondria, leading to programmed cell death and DNA damage.
Scientists have successfully used optogenetics to control seizure activity in living human brain tissue, opening doors to new treatments for epilepsy and other neurological diseases. By switching off specific neurons with light pulses, researchers can prevent seizures from occurring, providing a less invasive alternative to surgery.
The institute's Spark Grant program recognizes innovative research in gene and cell therapy, with recipients working on projects spanning disease fields such as neuroinflammation, cancer, and tuberous sclerosis. The $1.15M grant will support the development of novel treatments and commercialization outcomes.
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%.
Scientists from Trinity College Dublin have developed a gene therapy that protects retinal ganglion cells and improves their function in animal models of glaucoma. The therapy has also been shown to increase oxygen consumption and ATP production in human retinal cells, indicating enhanced cell performance.
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 developed a treatment approach using lipid nanoparticles to deliver mRNA therapy for MSUD, extending survival and reducing serum leucine levels. The study also identified a novel AAV variant with desirable biodistribution properties for targeting peripheral organs.
Researchers at the Broad Institute of MIT and Harvard developed a machine-learning approach to design better AAVs for gene therapy. The tool helps engineer capsids with multiple desirable traits, such as targeting specific organs or working in multiple species. About 90% of predicted capsids successfully delivered cargo to human liver ...
Researchers found that subretinal adeno-associated virus 8 injections trigger proinflammatory T-cell responses, but co-injection of immunodominant peptides can modulate the immune system. This study suggests a new approach to AAV gene therapy for retinal diseases.
Researchers at UW Medicine have developed a new gene therapy that delivers protein packets to replace defective genes in muscles, halting disease progression and reversing pathology. The therapy uses adeno-associated viral vectors and aims to restore normal muscle health, with human trials expected to begin in two years.
Researchers describe redundant innate immune pathways triggered by AAV vectors, including sensing of viral genome and cytoplasmic DNA sensors. The study highlights the need to understand complex biologic mechanisms underlying adverse reactions to AAV vectors in human gene therapy trials.
A novel gene therapy has restored hearing function in five children with DFNB9 who were treated in both ears, demonstrating additional benefits compared to a previous trial. The treatment also improved speech perception and sound localization abilities, including the ability to appreciate music.
ConVERGD, a new tool developed by St. Jude Children's Research Hospital scientists, enables precise manipulation of cellular subpopulations for studying specific behaviors. This breakthrough technology has the potential to revolutionize fundamental research and healthcare.
Researchers are developing CRISPR-Cas gene editing technology to modify and attack AMR bacteria, offering new tools to battle the increasing rates of antimicrobial resistance. By targeting specific genes and using phage-based delivery systems, scientists aim to develop safer therapies.
Researchers at University of Pittsburgh are developing a platform to genetically modify glia cells using bioengineering modified RNAs. The goal is to increase or decrease disease-relevant genes in astrocytes or microglia to potentially treat Alzheimer's disease and other neurodegenerative disorders.
Scientists have successfully tested novel gene therapies in a whole human liver, opening up new avenues for treating life-threatening inherited diseases. The use of a normothermic liver perfusion system enables accurate testing of gene therapeutics directly in the clinical target organ.
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 have found that a gene-based therapy targeting plakophilin-2 can interrupt the progression of arrhythmogenic right ventricular cardiomyopathy, a rare inherited disorder. The treatment reduced episodes of arrhythmia and slowed the deterioration of the heart's walls in mice.
Researchers at Duke University successfully introduced a mutated BRAF gene into rat heart tissue, inducing growth and cell division. However, the approach was associated with significant loss of contractile strength, highlighting the need for precise control over gene activation and delivery.
Researchers at NTNU are developing a new therapy for rare hereditary diseases like DOOR syndrome using mini-brains grown in the lab. The treatment involves injecting a virus with a healthy OXR1 gene to produce the missing protein, which may help stop or reverse the disease.
Researchers have developed a novel approach, REVeRT, to efficiently transport large genes using dual AAV vectors at the transcript level. This new method offers increased efficiency, fewer side effects, and greater flexibility compared to existing strategies.
Researchers created a new CRISPR-based gene therapy tool using locally sourced, human-derived proteins that can activate silent or insufficiently expressed genes. The DREAM tool mimics the natural ability of human cells to turn on specific genes in response to mechanical cues.
Researchers at Rice University have developed a non-invasive gene delivery technique using ultrasound to efficiently deliver clinically used gene therapy vectors throughout the brain. The study, published in Gene Therapy, shows that opening more sites within targeted regions improves gene delivery efficiency.
Researchers at Oregon Health & Science University have found that implanting a specific molecule in the brain can effectively reduce alcohol use among chronic heavy drinkers. The study showed a 90% reduction in drinking behavior in nonhuman primates, with the implanted virus permanently increasing dopamine levels and reducing cravings.
Researchers have developed a new gene-editing tool that enables the precise study of single-base mutations in cancer-causing genes. This technology allows for detailed experiments in tissues, which may lead to better understanding of genetic changes influencing patient response to cancer therapies.
Researchers at EMBL Heidelberg discovered that mutations in the RBM20 gene cause familial DCM by disrupting normal RNA splicing, leading to detrimental cytoplasmic granules. Targeted gene editing via CRISPR-Cas9 and restoring nuclear localisation of RBM20 could improve therapy options for patients.
Researchers successfully delivered stabilized divalent siRNA molecules to animal models that blocked SARS-CoV-2 and prevented infection. The technology is adaptable for other pulmonary diseases such as pulmonary fibrosis and respiratory viruses.
Researchers developed a translational step forward in treating brain tumors using intraventricular immunovirotherapy, which has shown safety and efficacy in recent clinical trials. This approach uses oncolytic herpes simplex virus type-1 to target high-grade glioma with promising results.
Scientists at Duke University have made a breakthrough in controlling gene expression in response to injury, using a segment of fish DNA called TREE. The method successfully targeted gene activity to specific regions and time windows, showing promise for regenerating damaged tissues in mammals.
Isaac Hilton is using non-integrating episomal DNA viruses to create a new platform technology for cell and gene therapies. He aims to hijack these viruses to safely program medicinal functions in human cells.
Nanoscope Technologies has developed a novel, non-viral gene delivery approach using low-power near-infrared lasers to target the retina. The method sensitizes cells to ambient light, improving visual response and offering a unique therapy option for dry-AMD patients.
A novel gene therapy strategy using hyperbranched poly(amidoamine) nanoparticles has been developed for cardiac repair. The strategy, known as HRE-VEGF, provides a safer alternative to current VEGF gene delivery systems and shows promise for treating myocardial infarction.
Researchers have developed a novel method for gene delivery using steroid-coated DNA, which improves cell uptake and reduces inflammatory immune responses. The technique has the potential to enhance gene transfer in humans, especially in inflamatory diseases.
A new study by UCSF researchers found that delivering the VEGF growth factor into mice with coronary heart disease prompted the growth of blood vessels in damaged heart tissue without causing side effects. This approach could lead to a treatment strategy for severe coronary heart disease that has been met with setbacks.
A new gene therapy technique using stents with DNA delivery has shown promise in animal studies for treating coronary artery disease. The method uses a biodegradable film to release genes into cells on the artery wall, inhibiting cell growth and potentially preventing further damage.
Researchers at Thomas Jefferson University have developed a new gene therapy system that uses DNA and bacterial proteins to deliver genes to specific places in human DNA, avoiding viruses and their associated immune reactions. The technique shows promise for treating genetic diseases such as hemophilia and sickle cell anemia.
Researchers at Duke University Medical Center have successfully used a heat-sensitive gene to slow down tumor growth in mice by 300-400 times over baseline. The gene therapy, which uses hyperthermia, also boosts the immune system's ability to fight cancer.
University of North Carolina at Chapel Hill scientists have discovered a receptor molecule that can facilitate the introduction of corrected genes into cell nuclei, paving the way for potential gene therapy treatments. The study uses a G-protein-coupled receptor to enable weakened viruses to enter cells, carrying therapeutic payloads.
Researchers at University of Iowa have found that a specific gene vector, AAV5, can effectively reach many brain sites following a single injection. The study suggests that AAV5-based vectors could be used to deliver correct copies of genes to cells throughout the central nervous system without multiple injections.
Researchers at Thomas Jefferson University have used gene therapy to replace a damaged Rb2 gene with a healthy version, dramatically shrinking lung cancer tumors in mice. The study provides direct evidence that Rb2 is a tumor suppressor and demonstrates its potential as a target for human gene therapy trials.
Researchers at the University of Washington discovered that HIV alters gene function in cells within three days of infection, affecting T-cell signaling and immune system functions. The study used cDNA microarray technology to examine gene expression levels and may lead to new treatments for the disease.
Researchers are testing gene therapy using nerve growth factor (NGF) to slow Alzheimer's disease progression. The trial aims to assess the safety of NGF-producing cells injected into patients' brains, with potential benefits for slowing dementia.
Researchers at Thomas Jefferson University found that a tumor suppressor gene, Rb2/p130, can halt rapid cell growth and prevent smooth muscle cells from building up in arteries. This gene may be used as an adjunct to angioplasty to treat clogged coronary arteries and reduce the risk of restenosis.
Researchers have successfully delivered a therapeutic gene for peripheral neuropathy using a modified herpes virus, which can produce nerve growth factor (NGF) in non-neuronal cells and enter the bloodstream. This approach may offer a long-term solution for patients with peripheral neuropathy resulting from diabetes or chemotherapy.