Articles tagged with Gene Targeting
Smithies' groundbreaking work on gene targeting has led to thousands of mouse models simulating human diseases, enabling rapid advances in diagnosis and treatment. His research using genetically altered mice has also shed light on high blood pressure, a major human problem.
Researchers have identified a brain region involved in planning movement, which also controls gain control of eye movements. This finding provides new insight into how the brain adjusts to moving objects, allowing for smooth image display.
Researchers Beau Lotto and Dale Purves suggest that the visual system perceives color based on reflectances and illuminances of past stimuli. In experiments, subjects adjusted colors to match perceived color differences, showing an increased difference when contexts suggested different objects or illumination.
Phase I clinical trials show CCI-779's potential as a cancer treatment, inhibiting tumor growth with minimal side effects. The drug targets the mTOR pathway, which may selectively arrest the development of certain cancer cells.
Researchers have discovered two targets for a new generation of cholesterol-lowering drugs that should allow greater precision in managing cholesterol levels. A key compound, LG268, has been shown to completely block the absorption of cholesterol in mice, while also enhancing the production of reverse cholesterol transporters.
Dr. Oliver Smithies has received the International Okamoto Award for his groundbreaking work in hypertensive and arteriosclerotic research using molecular genetics. He pioneered gene targeting techniques that have been widely adopted by researchers worldwide, leading to a better understanding of human genetic diseases.
A Phase I study of aerosolized administration of tgAAV-CF showed efficient gene transfer throughout the upper airways of patients with mild CF lung disease. No neutralizing antibodies were detected, suggesting a good safety profile for further development.
The company presented Phase II data of tgDCC-E1A, a lead cancer therapy, which showed a 45% objective response rate in patients with recurrent head and neck squamous cell carcinoma. Preclinical studies also demonstrated the ability of E1A to sensitize tumors to radiation and chemotherapy.
New research from the HOT Study reveals significant differences in the response to antihypertensive treatment between age and sex groups. Women with hypertension achieve target blood pressures more easily than men, while older patients exhibit improved blood pressure control and lower risk of adverse events.
A new prototype vaccine activates cytotoxic T-lymphocytes to destroy cancer cells using telomerase as a target. The vaccine has shown promise in both human and transgenic mice, indicating potential effectiveness against various types of cancer.
Researchers at UNC-CH have developed a gene therapy method that targets specific types of cells, avoiding the less effective 'shotgun' approach. The technique successfully corrects genetic defects in primitive stem cells, which can be used to treat inherited blood disorders like hemophilia and non-inherited illnesses such as cancer.
Scientists are using an old photographic technique to study nuclear physics and potentially develop a clean, long-term power source through thermonuclear fusion. The petawatt laser facility generates high-energy gamma rays that can be used to trigger tiny fusion reactions.
Researchers found that FTIs activate features of RHO by stimulating the production of an isoform with altered properties, making it possible for RHO to perform activities it could not previously perform. This understanding will lead to more effective and targeted cancer treatments.
A team of scientists has made a breakthrough discovery in understanding how the HIV virus attaches to immune system cells, revealing new targets for anti-HIV vaccines and drugs. The study found that the virus uses multiple defenses to evade attack, including shape-shifting projections and carbohydrate molecules.
Researchers demonstrate intratumoral delivery of the E1A gene, downregulating HER-2/neu expression and tumor responses. The study shows promise for using E1A gene therapy to treat a broad range of cancers.
Researchers successfully delivered and expressed an E1A gene in both diseased and normal human cells using a proprietary non-viral liposomal delivery mechanism. The study showed decreased levels of surrogate tumor markers in three patients with breast and ovarian cancers, indicating potential as a treatment for various types of cancer.
University of Washington researchers successfully use a modified virus to perform novel gene replacement, targeting mutated genes with high accuracy. The new method achieves efficient gene correction, allowing for precise control over the expression of the corrected gene.
Chemist Benjamin Miller has devised a way to create new drugs by using metal atoms to assemble countless combinations of molecules, then selecting the best candidates through a Darwinian process. This method shifts the burden of tedious drug development work off technicians' shoulders, offering a faster and more efficient approach.
Targeted alpha-particle therapy using Bismuth-213 has shown very encouraging results in leukemia patients, with no significant uptake outside the target areas. The therapy appears safe and feasible, with high linear energy transfer and reduced damage to normal tissue.
A team of researchers has developed a virus that controls HIV infection by targeting infected T cells. The modified virus, VSV, tricks infected cells into fusing with it instead of normal cells, allowing it to destroy the virus. This approach could lead to new treatments for HIV and other diseases.
A study published in Science journal demonstrates the efficacy of vascular targeting agents (VTAs) in treating large solid tumors. VTAs selectively occlude tumor vasculature, causing massive tumor cell death and advanced necrosis within 72 hours. The treatment is well-tolerated and results in complete or partial tumor regressions in mice.