Articles tagged with Gene Therapy
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.