Articles tagged with Gene Therapy
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.
Researchers have found that a gene therapy technique can reduce alcohol consumption in rats by increasing dopamine receptor levels. This approach may offer a new potential treatment for human alcohol abuse.
Researchers have successfully used gene therapy to reduce new blood vessel growth by up to 90% in mice with conditions similar to macular degeneration and diabetic retinopathy. Two genes, endostatin and pigment epithelium-derived factor (PEDF), were injected into the animals' eyes or tails to demonstrate the potential of gene therapy a...
Researchers developed a gene therapy that increased D2 receptor levels in rats, leading to a significant drop in alcohol intake. The treatment showed promise in reducing drinking preference and behavior in both groups of rats.
Researchers at UCSD will team up with Celera Genomics to identify and genotypic genetic variations in patients with hypertension, aiming to develop individualized treatment plans based on genetic profiles. The goal is to predict therapeutic drug responses and minimize side effects.
Researchers used gene therapy to treat mice with beta-glucuronidase deficiency, a condition that affects approximately 1 in 5,000 babies. The treatment showed promise by improving symptoms for up to a year, but six mice eventually developed cancer, raising questions about the long-term safety of this approach.
Scientists have produced new adult neurons from precursor cells using a growth factor, which could potentially treat neurological diseases. The technique, supported by the NIH, involves introducing brain-derived neurotrophic factor (BDNF) into the adult rat brain.
Scientists have identified an important parallel between C. elegans and human NMD pathways, revealing a potential therapeutic strategy for masking genetic mutations. Inactivation of the human homolog of the C. elegans smg-1 gene inhibits NMD, allowing truncated proteins to accumulate and potentially compensate for genetic disorders.
Research reveals heritability plays a major role in determining the age of menopause, with significant implications for women delaying childbirth beyond 30. Genetic analysis suggests that a woman's store of eggs declines throughout life, triggering menopause.
The complete genome of pneumococcus has been sequenced, revealing its genetic makeup and potential applications in treating the bacterium. The newly released genome contains 2,326 sequenced genes, providing a comprehensive model for researchers to study its virulence and develop new therapies.
Researchers tested four viruses to transport genetic material into defective eyes of 18-day-old rats. Adenovirus proved the most effective vector, targeting specific tissues and preventing disordered blood vessel growth. Early-stage research suggests a potential breakthrough in treating retinopathy of prematurity with gene therapy.
Jefferson researchers successfully introduced 90 billion copies of a healthy gene into the brains of three children with Canavan disease. The goal is to increase enzyme production and reduce toxic compound buildup, potentially improving brain development and function.
Researchers warn that angiogenesis therapy could trigger growth in abnormal tissue, increase artery-clogging plaque, and stimulate inflammatory response. Despite these potential complications, the authors remain hopeful that the therapy will ultimately prove effective and safe.
Dr. Boucher is a leading researcher on cystic fibrosis, publishing over 300 articles and developing novel treatments. The University of North Carolina's genomics initiative receives $245 million in funding to advance research and therapies.
Dr. Jeffrey S. Chamberlain joins the University of Washington to study muscular dystrophy gene therapies with a focus on developing vectors for genetic delivery. His research aims to prevent and reverse the disease, offering hope for treatment or cure options.
Scientists at UNC Health Care have successfully used gene-therapy to produce high amounts of a clotting protein in animals, bypassing a major hurdle for treating hemophilia. The new method could potentially yield sustained factor IX production in humans, eliminating the need for daily injections.
Researchers developed a tissue culture system that mimics normal cell growth, allowing them to study gene transfer techniques in greater detail. They found that cancer cells can be easily infected while normal cells are poorly infected, suggesting a potential target for gene therapy.
Researchers at Jefferson Medical College successfully introduced a therapeutic gene into the brain of two children with Canavan disease, showing decreased NAA concentration and increased myelin. The gene transfer demonstrated positive neurological improvements in the patients, offering hope for future use of gene therapy for brain dise...
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.