Researchers have identified a gene associated with inherited primary pulmonary hypertension, providing new avenues of study for determining its molecular basis. The discovery opens up possibilities for designing more effective therapies for this devastating condition, which affects primarily women of childbearing age.
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.
A genetically stripped down HIV-based vector has been developed that can deliver genes to target cells in the body safely. The vector can also be turned off with a common antibiotic, which suggests doctors may one day be able to control gene expression in people treated with HIV-based gene therapy vectors.
Dusty Miller, PhD, received the National Hemophilia Foundation's 1999 Dr. Murray Thelin Researcher of the Year Award for his work on gene therapy, a potential treatment for hemophilia. His pioneering research has led to the development of retroviral gene-transfer kits and promising early-stage gene-therapy trials.
Targeted Genetics Corporation has shown promise in its AAV gene therapy program for rheumatoid arthritis (RA), reducing ankle and hind paw swelling in preclinical studies. The treatment uses a recombinant TNFR:Fc fusion protein, which antagonizes the function of TNF-a, a key component of the inflammatory response.
Introgen presents Phase I trial results on RPR/INGN 201, showing safety and expression in ovarian cancer, lung carcinoma, and glioblastoma patients. The therapy has demonstrated significant potential in treating these life-threatening conditions.
Researchers conducted a Phase II trial of adenoviral p53 gene transfer with radiation therapy, showing 29% of patients achieved local tumor control and 12 of 15 had no active cancer. The study demonstrated considerable promise for combined treatments, comparing favorably to a control rate of less than 20% in patients treated with radia...
The JDF Center aims to develop new gene therapy techniques to increase islet cell supply for transplantation and protect transplanted cells from immune system attack. The center will focus on making islet cell transplants viable for young patients with Type 1 Diabetes.
The 108th Annual Convention will examine the role of psychology in changing healthcare environments, online therapy, and the human genome project. Keynote speakers Pete Seeger and Arthur Jensen will discuss their work on music, spirituality, and genetics.
Chemists at Washington University in St. Louis have created knedel nanoparticles that mimic viruses and show potential for a new direction in gene therapy and other biomedical applications. The nanoparticles can escape detection by the immune system and are designed to behave like viruses, but without the risk of live virus effects.
Researchers found a 28% decline in brain network density with normal aging in monkeys, which was reversed by transplanting genetically programmed nerve growth factor-producing cells. This approach may be useful for treating Alzheimer's disease, with clinical trials underway.
French researchers develop gene therapy to treat human severe combined immunodeficiency (SCID) X1, a life-threatening disease. Two infants experience 'striking' clinical improvements and return to normal immune system functioning without side effects.
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 report encouraging early results in a novel gene therapy designed to improve the clinical course of hemophilia B, with two patients requiring significantly less infused clotting factor to treat symptoms. No adverse side effects were experienced by any of the patients treated.
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 found that patients who received the gene therapy had less than half of the failures of those without the treatment, with improved vein wall adaptation and reduced blockage rates. The study suggests a new treatment approach to vastly improve bypass surgery success rates.
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.
Researchers from Children's National Medical Center and University of Pittsburgh successfully reverse muscle damage caused by limb girdle muscular dystrophy using gene therapy. The non-toxic virus-based approach increases muscle strength and size by nearly 100% in animal tests, paving the way for potential treatment of Duchenne muscula...
Researchers at Johns Hopkins Medicine successfully used gene therapy to activate the human immune system against metastatic prostate cancer. The study, published in Cancer Research, showed that patients' B-cells produced antibodies and T-cells directly attacked tumor cells after vaccination.
Researchers have discovered a genetic on-off switch in yeast that could be used to control insulin levels in diabetics. The switch, consisting of two main parts, can be turned on or off by adding a chemical inducer, allowing for tight control over gene expression.
Researchers at Northwestern University have halted motor neuron destruction and slowed ALS progression using GDNF gene therapy in laboratory mice. The approach may one day prove effective in treating humans with ALS.
New gene therapy study in mice demonstrates a gradual increase in factor VIII levels, stable for 11 months. The goal is one dose, which could be all that's needed to be physiologically cured of future bleeds.
Researchers at UPMC have successfully conducted the world's first in vivo gene therapy for hemophilia A using a modified retrovirus to deliver the FVIII gene. The treatment aims to permanently restore clotting factor production, reducing costs and complications associated with the disease.
The American Society of Gene Therapy is hosting its 2nd Annual Meeting, featuring key researchers and NIH director Harold Varmus. The event showcases the latest advancements in gene therapies for various genetic disorders, including AIDS, cardiovascular disease, cancer, cystic fibrosis, and neuromuscular diseases.
Researchers successfully produce widespread transfer of corrective genetic material into muscle cells using a naturally-occurring hamster model. The technique, developed by Penn researchers, overcomes the existing problem of accessing millions of muscle cells requiring genetic re-engineering.
Researchers successfully modified adenovirus, a common cold virus, to carry corrective genes to defective cells. The modified virus persisted for over two months in mice, overcoming a major barrier to widespread use of adenovirus as a genetic delivery vehicle.
Researchers successfully treated genetically modified mice with Parkinson's disease using gene therapy, restoring motivation to eat and drink. The study suggests a potential treatment strategy for patients with Parkinson's disease, eliminating the need for daily L-dopa treatments.
University of Florida researchers have successfully developed a gene therapy approach that replaces a crucial protein protecting the lungs from an often-fatal lung-liver disease. The method uses an adeno-associated virus to incorporate the missing protein, achieving high levels in the bloodstream for therapeutic effect.
Scientists have developed a novel gene therapy system that uses a molecular rheostat to control the activity of therapeutic genes. This system allows for precise dosage control and can be achieved through oral administration of a simple pill.
A study led by Ohio State University found that high-dose cytarabine increased the cure rate of acute myeloid leukemia (AML) patients with core binding factor AML to nearly five times, with a 78% remission rate among those receiving the treatment. The standard dose resulted in only 16% remission rates
Gene therapy aims to treat unborn children with genetic disorders like SCID and alpha-thalassaemia. The treatment involves introducing healthy copies of the defective gene into the fetus, but raises concerns about potential risks and long-term consequences.
Scientists at Thomas Jefferson University have demonstrated that a normally protective anticancer gene can block tumors from growing. Led by Dr. Antonio Giordano, they found that the damaged version of the gene, pRb2/p130, suppresses tumor growth in animal models, paving the way for gene therapy to treat lung cancer and other cancers.
Scientists have successfully administered gene therapy orally, effectively curing lactose intolerance in rats. The study demonstrates the potential for an oral delivery system to treat other diseases, such as diabetes.
Researchers have successfully used gene therapy to slow down neurodegeneration in an animal model, offering hope for treatments of autosomal dominant disorders. The approach involves using ribozymes to destroy faulty messenger RNA, potentially treating conditions like Huntington's disease and ALS.
Researchers at Thomas Jefferson University used antisense therapy to prevent Burkitt's lymphoma development in laboratory mice, delaying tumor onset and decreasing its size. The strategy may also work in human patients who have relapsed after chemotherapy, with potential applications against various types of cancer.
A University of Florida neuroscientist has discovered a mutant gene in chickens that helps researchers better understand a rare human eye disease called Leber's congenital amaurosis type I. The chicken model is being used to develop a form of gene therapy for treating the disease, which affects 100,000 to 200,000 people worldwide.
Scientists at UNC Chapel Hill and Bern University have developed a new RNA repair technique to block defective processing in cells' messenger RNAs. This method has shown promising results in increasing healthy protein production and could potentially treat or even cure beta thalassemia.
Researchers found that localized gene therapy can produce widespread, systemic effects on rheumatoid arthritis. The study used an adenovirus to carry therapeutic genes for altered cell receptors, which lessened inflammation and cartilage destruction in both diseased and distant joints.
The American Society of Gene Therapy will hold its inaugural annual meeting from May 28 through May 31 in Seattle, featuring sessions on gene therapy vectors and various diseases. Over 1,500 scientists are expected to attend, with a focus on education and collaboration.
A 15-year study of 628 AML patients confirms that chromosomal abnormalities can predict treatment success and likelihood of cure. Patients with specific genetic markers tend to have better outcomes, while those without them face poorer survival rates.
Scientists at Ohio University and Progenitor Inc. use a nonviral gene expression system to eliminate human cancer cells in animals, achieving a 60 percent tumor regression rate. The T7 system allows for transient gene expression and has been shown to be effective against various types of cancers.
Researchers at Thomas Jefferson University are conducting a gene therapy trial for glioblastoma, a form of brain cancer. The trial aims to delay disease progression and improve quality of life for patients, offering a potential cure for the deadliest type of brain tumor.
Researchers successfully used gene therapy to grow new blood vessels in eight people with obstructed leg circulation due to atherosclerosis. The therapy, called therapeutic angiogenesis, helped improve blood flow, reduce pain and gangrene risk, and even prevented amputations.
A new form of gene therapy has been tested to prevent blockages that occur after bypass surgery, with four patients showing successful results. The treatment aims to reduce cell growth and atherosclerosis in grafted vessels, which can fail due to up to 50% of cardiac and leg bypass surgeries.
A study by scientists at NIAID found that gene therapy can result in prolonged production of functionally normal white blood cells in patients with chronic granulomatous disease. This finding has important clinical implications for the treatment of this rare immunologic disorder.
Researchers have developed a gene therapy technique that restricts the activity of genes used for gene therapy to specific cell types, bypassing safety hurdles. This discovery could lead to targeted treatment of cardiovascular diseases and other disorders.
Scientists at Case Western Reserve University have created artificial human chromosomes, offering a powerful tool for studying human genetics and potentially treating genetic diseases. The synthetic microchromosomes demonstrate normal centromeric activity, genetic stability, and continued gene expression.
Brown University researchers have developed a novel oral drug delivery system using erodible plastic microspheres that can improve the efficacy of drugs like insulin. The microspheres, engineered with bioadhesive polymers, increase absorption rates and enhance therapeutic effects.
Researchers at University of Pittsburgh Medical Center have made significant progress in treating rheumatoid arthritis using gene therapy. They successfully expressed the interleukin-1 receptor antagonist (IL-1Ra) gene in synovial cells within joints treated with the approach.
Researchers have developed a gene therapy technique that can produce and secrete proteins into the bloodstream indefinitely, without the need for viral delivery systems. This could drastically decrease the cost of treatments for certain types of anemia, which affect over 140,000 people in the US annually.