Articles tagged with Gene Therapy
New research from University of Minnesota presents promising findings on unrelated donor transplants, including a study showing improved survival rates and quality of life for leukemia/lymphoma patients. Another study explores the use of bone marrow-derived stem cells to treat Parkinson's disease.
Researchers have found that blocking myostatin in mice with muscular dystrophy improves muscle function, providing a potential new treatment for the condition. However, further studies are needed to address concerns about the approach's limitations and potential side effects.
Researchers have developed a novel system of gene therapy that targets lung stem cells to treat cystic fibrosis. By using a natural compound found in the lungs, the therapy enables airway cells to take up the therapeutic gene and shows substantial recovery from the disease defect.
As new genetic components are identified, clinical services must adapt to cope with the increasing demand. The role of genetic information in managing inherited cancers is becoming increasingly important, enabling tailored therapies for individual patients.
Researchers have developed a gene therapy technique that integrates DNA without using viral vectors, inserting the DNA at known locations. The technique successfully integrated a gene coding for Factor IX into mouse DNA, making 12 times more protein than control mice.
A new study using gene therapy has shown promise in treating Parkinson's disease by 'resetting' overactive brain cells. The therapy may arrest or delay disease progression, offering a safer and more powerful alternative to current treatments.
Researchers successfully used gene therapy to change the brain's circuitry in rats with Parkinson's disease, producing nearly 70% fewer symptoms. The treatment involves altering glutamate and GABA signaling pathways to protect dopamine neurons from deterioration.
A team led by Purdue University's David Sanders has developed a promising system to deliver genes to diseased liver and brain cells using a modified Ross River Virus (RRV) as a Trojan horse. This approach avoids the harmful side effects associated with other retroviruses, bringing gene therapy closer to treating human diseases.
Researchers at the University of Florida have demonstrated a novel approach to overcoming gene therapy hurdles by attaching viruses to tiny manufactured balls coated with iron oxide. The microspheres can be targeted using magnets, allowing for more efficient delivery of corrective genes to specific organs or tissues.
Researchers develop novel approach to treat thalassemias by blocking aberrant RNA splicing. The new virus effectively repairs existing RNA, restoring correct hemoglobin production and offering a lifeline for patients.
Researchers have successfully delivered the full-length dystrophin gene to mice with muscular dystrophy using stripped-down vectors, restoring normal muscle function. The breakthrough could pave the way for human clinical trials to assess the safety of this method in patients.
A novel gene therapy technique developed by Stanford researchers allows for the integration of large genes into human chromosomes, enabling continuous production of proteins. This technique has the potential to treat a variety of diseases, including Duchenne's muscular dystrophy and cystic fibrosis.
Researchers have developed a gene therapy approach to treat mucopolysaccharidosis VII, a disorder affecting multiple organ systems, in dogs. The treatment involves four intravenous injections of a retroviral vector expressing canine beta-glucuronidase, resulting in normal enzyme activity and near-normal mobility in treated dogs.
Researchers developed a gene therapy combining radiation therapy and cisplatin to target tumor cells, resulting in significant regression with no additional toxicity. The treatment uses a modified cold virus to insert the TNF gene into tumor cells, enhancing anti-cancer effects.
The study discovered a novel gene, ADAM33, linked to asthma susceptibility and airway changes in asthmatic patients. This gene is involved in generating bronchial hyperresponsiveness and may underlie abnormalities in asthmatic airway function.
A study identified three gene expression patterns correlated with survival after chemotherapy, which outperformed the International Prognostic Index. The patterns revealed aspects of tumor biology affecting treatment response, including immune system involvement and tumor cell division.
A new gene therapy protocol has successfully treated a child born without an immune system, reversing severe combined immunodeficiency. The procedure improved genetically altered stem cells, giving them a biological advantage and allowing them to prevail over the abnormal cells.
Researchers have successfully used gene therapy to treat two young children with ADA-SCID, a rare form of SCID that requires regular injections of the bovine form of ADA enzyme. The new method involves removing bone marrow cells and engineering them to produce healthy immune cells, offering a potentially lower-risk alternative to bone ...
Biologists found that nematodes use a sophisticated mechanism to render transposons harmless, preventing them from making proteins and jumping through DNA.
Researchers at Georgetown University Medical Center have developed a germ cell line derived from spermatogonia, offering a basic tool for studying male infertility and testicular cancer. This cell line exhibits characteristics of normal adult spermatogonial stem cells and differentiates into advanced germ cell types upon stimulation.
Researchers created a mouse model of hair loss syndrome, which sheds light on the complex interactions between genes and their effects on human diseases. The study found that genetic background plays a significant role in determining the severity of the condition, and could potentially inform gene therapy approaches.
A recent study, TRAFFIC, found that intra-arterial infusions of recombinant fibroblast growth factor-2 (rFGF-2) improved exercise capacity in patients with moderate-to-severe intermittent claudication. Peak walking time increased by 1.77 minutes with single-dose rFGF-2 and 1.54 minutes with double-dose rFGF-2.
A new gene therapy tool has shown promising results in treating mice with hemophilia A by correcting the faulty factor VIII gene. The technology uses RNA trans-splicing to produce normal factor VIII protein, offering a potential cure for this genetic disorder.
Dr. Katherine High, a leading gene therapy researcher, has been chosen by the Howard Hughes Medical Institute for her groundbreaking work on treating hemophilia B. Her research holds promise for delivering therapeutic genes directly into patients' cells to improve blood clotting and quality of life.
Researchers have discovered the location of the genes that can cause heart attack, a breakthrough that could lead to new means of prevention and treatment. The study used genetics maps to scan chromosomes and found regions on chromosomes two, three, and 20 linked with heart attacks.
Scientists have found a way to activate the utrophin gene, which can help compensate for the lack of dystrophin protein in muscles affected by Duchenne's muscular dystrophy. The study suggests that over-producing utrophin may be a viable alternative to adding a working copy of the dystrophin gene through gene therapy.
Research findings link insulin-like growth factor I (IGF-I) to decreased lung cancer risk when bound by IGFBP-3. Environmental factors influence DNA methylation patterns in tumor suppressor genes, which may impact cancer incidence. Electro-gene therapy enhances IL-12 protein delivery, triggering long-lasting antitumor responses.
Researchers have successfully used a virus that causes warts to restore LDL receptor function in cells with defective genes, offering new hope for treating familial hypercholesterolemia. The next step is testing this technique in animals and humans to determine its safety and effectiveness.
Researchers at UT Southwestern and M.D. Anderson have identified eight genes on chromosome 3 that act as tumor suppressors in lung cancer. These genes were found to inhibit tumor growth and induce cell death in human lung cancer cells, suggesting a potential new approach for treating the disease.
A new type of dendritic cell has been found to prevent diabetes in mice by causing T cells to die rapidly. In a study, researchers treated mice with this special dendritic cell and found that half remained diabetes-free at one year.
Researchers analyzed participants' CETP genes to find genetic markers for heart disease. They discovered a pattern of gene variations called haplotype, which showed a significant association with the person's response to statin therapy.
Researchers discovered two mechanisms by which adenoviruses establish persistent infections: interfering with apoptosis signaling and preventing apoptosis through the Receptor Internalization and Degradation complex. This knowledge could improve the usefulness of adenovirus vectors in gene therapy.
The Lancet Neurology aims to report the most important advances in neurology, predicting risk of neurological conditions through genetics and imaging. Experimental therapies like gene therapy and stem cells offer potential for treating neurological disorders.
A study by UC Berkeley's Bruce N. Ames found that megavitamin therapies can effectively treat over 50 genetic diseases, mostly rare metabolic disorders due to defective enzymes. High-dose vitamins, particularly B vitamins like niacin and thiamine, may also improve metabolism in older populations.
A team of researchers at the University of Pittsburgh has discovered a gene mutation responsible for hereditary gingival fibromatosis (HGF), a rare condition characterized by gum overgrowth. The discovery may lead to new therapies, including tissue engineering, and could have implications for treating common dental problems like period...
Researchers successfully treated rats with chemical-induced Parkinson's disease using gene therapy, restoring normal limb movement. The treatment involves delivering two corrective genes into a specific brain region to prime the production of dopamine, which coordinates limb movements.
Researchers found that women with a common mutation in the estrogen receptor alpha gene had dramatic increases in high-density lipoprotein (HDL), or the 'good' cholesterol. This discovery suggests that genetic testing may help doctors make better decisions about hormone replacement therapy for preventing heart disease.
Researchers have discovered at least 100 genes that show significant differences in activity between non-invasive ductal carcinoma in situ (DCIS) and invasive breast cancer (IBC). The study's findings suggest that defects in these genes may play a role in the progression from non-invasive to invasive cancer.
A new intracoronary gene therapy has shown promising results in treating stable angina, a condition affecting nearly 7 million people nationwide. The study, led by the University of Vermont, involved 79 patients and demonstrated improved exercise ability compared to placebo groups.
Researchers successfully combined therapeutic cloning, embryonic stem cell differentiation, and gene therapy to treat a genetic immune disorder in mice. The study demonstrates the potential for nuclear transplantation therapy to correct genetic mutations and restore function in human patients.
Researchers have identified key genes associated with cardiac hypertrophy, hypertension, and dilated cardiomyopathy. Genetic testing is also being developed to predict susceptibility to coronary artery disease, offering new avenues for preventive medicine.
Gene therapy has shown promising results in hemophilia trials, with patients experiencing significant improvements in quality of life. The treatment involves inserting a normal version of the defective gene to produce clotting factor, and has been tested in small animals before being moved to human trials.
Researchers at Northwestern University have developed a gene therapy that reduces inflammatory cytokines involved in joint inflammation. The treatment, which uses the IL-13 gene, has been shown to suppress joint inflammation and prevent the development of arthritis in rat models.
Scientists have identified the PKHD1 gene as the cause of autosomal recessive polycystic kidney disease (ARPKD), a life-threatening genetic disorder. The discovery provides a genetic roadmap for understanding how the disease progresses, paving the way for more accurate diagnoses and treatments.
Researchers have developed a gene therapy using human blood outgrowth endothelial cells to treat hemophilia A. The treatment, which has shown long-term attainment of robust levels of factor VIII, is promising and may pave the way for testing in humans.
A researcher at Medical College of Georgia has identified three genetic flaws on chromosomes in patients with the most common type of Alzheimer's disease. Dr. Poduslo's study suggests that late-onset Alzheimer's is linked to multiple genes and may be subdivided into specific categories, leading to improved diagnosis and treatment options.
Researchers used Sangamo's ZFP technology to repress a specific gene variant, preventing fat cells from developing. This breakthrough demonstrates the precision of ZFPs in regulating gene expression and may lead to new treatments for diseases related to fat cell development.
The American Society for Neuroregeneration and Transplantation (ASNTR) has published guidelines to ensure the development of novel cell and gene replacement therapies for brain disorders. The recommendations focus on preliminary study design, clinical trial methodology, and conflict of interest disclosure.
A new gene therapy method has successfully corrected sickle cell disease in mice by transferring an anti-sickling gene to bone marrow, preventing the formation of deformed red blood cells. The therapy, developed using a viral delivery system, resulted in up to 99% expression of the new gene in circulating red blood cells.
The UNC-Chapel Hill Gene Therapy Center will use the funds to translate basic research knowledge into safe human clinical trials, with a focus on cystic fibrosis and hemophilia. The center aims to provide new treatments for diseases caused by single-gene defects.
Scientists at Uppsala University have identified a gene that regulates mast cell survival upon allergic activation, providing hope for a novel therapy to treat allergies. Inhibition of this gene, A1, leads to the death of mast cells, decreasing their numbers and alleviating allergic symptoms.
Researchers led by Robert E. Braun analyzed gene expression after adenovirus delivery using both in vitro and in vivo studies. The study found no infection of reproductive stem cells, supporting the safety of adenovector use in cardiovascular therapy.
Researchers genetically engineered mice to overexpress the HIF-1α gene in skin cells, resulting in a 70% increase in capillaries and normal-like blood vessels. The study suggests that HIF-1α harnesses VEGF expression in a way that could be beneficial therapeutically for conditions like ischemic tissue and recalcitrant wounds.
Researchers at Women's and Children's Hospital in Adelaide have developed a way to safely use Human Immunodeficiency Virus Type 1 (HIV-1) to transfer therapeutic genes into human cells. This method has wide applicability for various human genetic diseases and is currently being tested on animal models before moving to human trials.
A systematic study confirms the potential of pharmacogenomics to improve drug therapies by selecting treatments based on a patient's genetic profile. However, the practice faces significant challenges, including inadequate training, funding, and concerns about racial inequities.
In two studies, VEGF gene therapy significantly reduced angina episodes and improved exercise tolerance in patients with end-stage coronary heart disease. The treatment has shown impressive clinical results, with most patients experiencing continued improvement in daily life after treatment.
A new 'double suicide gene' therapy has shown promise in treating prostate cancer by carrying pairs of fused genes directly into cancer cells and inducing self-destruction. The technique achieved "greater levels of targeted cytotoxicity" than single suicide gene therapy, with minimal toxicities reported among patients.
Recent research highlights the significance of sodium-calcium exchange in various physiological processes, including vision, secretion, and cardiac contractility. The study also explores its potential applications in cancer therapy and heart function.
Researchers at Brookhaven National Laboratory discovered that coxsackievirus forms pairs on the surface of human cells, increasing the likelihood of infection. The study reveals hidden binding sites on the virus that evade the immune system, making it hard to defeat.
Researchers at UC Berkeley have discovered a single gene that can stimulate the immune system to reject a range of cancer tumors. The gene, which activates natural killer cells and other immune cells, shows promise as a potential basis for a vaccine therapy.