Articles tagged with Gene Therapy
Researchers have discovered a way to regulate gene therapy for Parkinson's disease by introducing an 'off switch' using the antibiotic doxycycline. This breakthrough addresses safety concerns and paves the way for safer treatment options.
A $3.5-million NIH grant will help advance a gene therapy for Tay-Sachs disease from animal tests to human clinical trials. The Boston-based consortium has successfully paired two genes in a single vector, which they believe will increase therapeutic efficiency and lower production costs.
Parkinson-associated genes PINK1 and Parkin functionally interact to maintain mitochondrial function, impairing neuron loss
Researchers found that HPRT gene plays crucial role in regulating transcription factors' expression and development of neurons, supporting a possible link to Lesch Nyhan disease. The study provides first direct experimental support for HPRT's involvement in neurological disorders.
MU researchers discovered a 'longevity' gene that energizes mice during exercise, potentially applicable to humans. The gene removes toxic free radicals from mitochondria, improving life quality and treating diseases like muscular dystrophy and diabetes.
Researchers report significant visual benefits in three young adults with Leber congenital amaurosis, a previously untreatable form of blindness. The gene therapy, which restored the RPE65 protein necessary for vision production, has shown safety and stability over one year.
Researchers have documented remarkable vision improvements in three young adults with Leber Congenital Amaurosis after receiving gene therapy. The patients continue to experience increased sensitivity to light, and one patient was able to read an illuminated clock for the first time at 1 year after treatment.
Researchers have discovered that gene therapy can restore vision in adults with Leber congenital amaurosis type 2, a rare form of blindness. The treated parts of the retina have acquired image-processing strength to rival the normal center for visual perception.
Researchers at University of Florida have safely given new genes to patients with alpha-1 antitrypsin deficiency, enabling them to produce trace amounts of the protective protein for up to one year. This potential step toward a gene therapy could benefit approximately 100,000 Americans with the condition.
Researchers successfully delivered functional genes to patients with alpha-1 antitrypsin deficiency, triggering the production of a protective protein. The study shows promise for gene therapy as a potential treatment option for this genetic disorder.
Researchers at the University of Leeds have successfully prevented epilepsy in mice by identifying a faulty gene as the cause. The study, published in PNAS, offers irrefutable proof that a defective version of the Atp1a3 gene is responsible for causing epileptic seizures in mice.
A study of nearly 1,600 tumor samples found that CT-X genes are expressed in half of ER-negative and triple-negative breast cancers. This suggests a potential new therapy approach for these aggressive breast cancer types. Researchers plan to initiate clinical trials based on the findings.
A research team has used single-molecule fluorescence microscopy to follow magnetic nanoparticles as they transport genes or drugs into target cells in real-time. The study reveals bottlenecks in nanoferry transport and provides insights into optimizing existing systems, potentially leading to breakthroughs in gene therapy.
Researchers at UNC School of Medicine have found an efficient way to deliver a corrected gene to lung cells, restoring normal function in tissue models. This breakthrough could lead to improved treatment options for cystic fibrosis patients.
Researchers at University of North Carolina have discovered a new approach to delivering a corrected CF gene to lung cells using parainfluenza virus, which could potentially cure cystic fibrosis lung disease. The method has shown promise in laboratory models and may pave the way for future clinical trials.
Researchers developed an ideal strategy for developing HCV-specific gene therapy by identifying the OAS promoter as a potential target. The study demonstrated that the HCV-core protein activates the OAS promoter specifically and effectively, paving the way for potential targeting of HCV using this approach.
A recent study published in JAMA Network has found a network of altered genes that appear to play a crucial role in the development and progression of brain tumors. The researchers identified seven landscape genes that are associated with patient prognosis and survival.
Researchers at Cedars-Sinai have developed a novel gene therapy that uses immune cells to target glioblastoma multiforme. A specific biomarker, HMGB1, has been identified as an effective tool to monitor tumor response to this treatment.
Scientists have made significant discoveries in the fight against muscular dystrophy, identifying a potential gene therapy approach to overexpress Galgt2 protein. In another breakthrough, researchers have identified a stromal marker for breast cancer progression, highlighting the importance of examining stroma PDGF receptor expression ...
A TGen-led team has identified five genetic biomarkers that may predict response to the anti-diabetes drug Actos, enabling personalized medicine for patients with type 2 diabetes. These markers were found in genes associated with PPARG function and include variants in a key drug metabolizing gene called cytochrome P450 3A4.
Researchers have developed a gene delivery approach to target therapeutic genes to nerves in the dorsal root ganglion (DRG), a region affected in various sensory neuronopathies. This method, using helper-dependent adenoviruses, was found to be more efficient at delivering genes to DRG nerves compared to nontargeted versions.
Researchers discovered that delivering a small molecule that is highly expressed in normal tissues but lost in diseased cells can result in tumor suppression. The study employed a novel strategy to treat hepatocellular (liver) cancer, using microRNAs (miRNA) gene delivery via a recombinant adeno-associated virus (AAV).
Researchers develop synthetic protein that induces blood clotting, cutting off tumor's nutrient and oxygen supply. The treatment resulted in a 53% decrease in tumor volume and slowed cancer cell growth by 49%.
Researchers at the University of Minnesota Medical School have successfully provided long-term expression of Factor VIII in hemophilia A mice using a new gene therapy approach, marking a promising step toward human clinical trials.
Researchers develop gene therapy approach to target Factor VIII in hemophilia A mice, reducing disease symptoms. L-Asparaginase degradation mechanism also uncovered, with potential for optimizing treatment.
A team of researchers identified astrocyte elevated gene-1 (AEG-1) as a critical gene that regulates tumor progression in neuroblastoma, a form of cancer commonly found in young children. Loss of AEG-1 was shown to reduce the tumor-causing properties of aggressive neuroblastoma cells.
A team of researchers has successfully developed a combined stem cell-gene therapy approach that cures human Fanconi anemia, a genetic disorder causing bone marrow failure and cancer. The treatment uses induced pluripotent stem cells to differentiate into healthy blood cells, offering a potential cure for the disease.
Researchers at UF have developed gene therapy that restores the faulty enzyme for glycogen storage disease type 1A, allowing dogs to survive for extended periods without glucose supplementation. The treated dog, born with the deadly disease, is now 20 months old and healthy after receiving precise doses of cornstarch every few hours.
Researchers have discovered a new therapy that substitutes the missing protein dystrophin to repair weakened muscle tissue in mouse models with Duchenne muscular dystrophy. The TAT-utrophin-based protein approach addresses the cause of the disease without gene replacement or stem cell issues, offering a potential treatment option.
A study by University of Florida researchers suggests that the nervous system may be the primary cause of severe breathing problems in children with Pompe disease, a rare genetic disorder. The discovery shifts responsibility from muscle weakness to signaling between the spinal cord and the diaphragm.
Researchers discuss a combined approach to maximize the benefits of stem cell therapy while minimizing risks. Gene therapy has the potential to solve the problem of wayward stem cells wreaking havoc after transplantation by genetically modifying them prior to use.
INTUNIV demonstrated significant efficacy in treating ADHD symptoms, particularly in children aged 6-12 years with oppositional behaviors. The study showed improved symptom reduction on various rating scales, including the ADHD Rating Scale-IV and Clinical Global Impressions-Improvement.
Researchers at Fox Chase Cancer Center have discovered a genetic pattern that can help predict how patients with gastrointestinal stromal tumors respond to the targeted therapy imatinib mesylate. By analyzing tumor specimens, they found genes that could be suppressed to enhance the effectiveness of imatinib therapy.
Researchers have identified a genetic signature that can predict which breast cancer patients will respond well to treatment with epirubicin, a widely used form of chemotherapy. This signature may help ensure fewer women are exposed to severe side effects by identifying those most likely to benefit from treatment.
Researchers at Yale University have developed a novel approach to deliver small interfering RNA (siRNA) molecules using biodegradable nanoparticles, achieving sustained release and effective knockdown of gene activity. This breakthrough holds promise for the treatment of sexually transmitted diseases (STDs), particularly HPV and HIV.
Researchers at VIB develop efficient and safe gene delivery approach based on non-viral genetic elements called transposons, overcoming limitations of viral vectors. This technology may simplify gene therapy, improve safety and reduce costs, treating cancer and genetic disorders.
Scientists at the University of California, Berkeley and Los Alamos National Laboratory have developed a comprehensive numerical study of gene therapy. The research sheds light on the key factors that determine the success of dendrimers as gene delivery vehicles, including the charges of the dendrimers and their target cell membranes.
Research found that women with tumors carrying TOP2A gene alterations had better outcomes when treated with anthracycline-based regimens, similar to those seen in HER2-positive tumors. The study suggests that TOP2A gene alteration is associated with increased benefit from anthracycline therapy.
The 157th ASA Meeting explores various disciplines and unique sounds. Wimps hear dangerous noises differently due to physical fitness, while fish living in darkness have exceptionally sensitive ears. Researchers also study medical devices using sound to treat disease, a long-gone whale's comeback, and cowards' evolution.
Researchers at Fox Chase Cancer Center identified a genetic pattern that may predict how patients respond to imatinib mesylate, and found genes that could be suppressed to enhance therapy effectiveness. The study's findings suggest potential new strategies for improving treatment outcomes in gastrointestinal stromal tumor patients.
Researchers at M. D. Anderson Cancer Center discovered genetic variations in the inflammation pathway that reduce bladder cancer recurrence and increase survival. Patients with risk-reducing genotypes had a 84% lower likelihood of disease recurrence after BCG treatment.
Researchers successfully transferred a gene to produce a protein necessary for healthy muscle fiber growth, increasing muscle-fiber size and producing the needed protein for at least six months. The findings demonstrate the feasibility of gene therapy for treating limb-girdle muscular dystrophy and other muscle disorders.
Researchers at the University of Michigan have developed a gene therapy approach that safely regenerates gum tissue. The method uses a localized application of adenovirus to deliver genes directly to the affected area, reducing the risk of systemic reactions.
Researchers suggest that autism symptoms may be reversible due to dysregulation of the locus coeruleus, a bundle of neurons processing sensory signals. The new theory proposes that fever can stimulate this system, potentially restoring its normal function.
Researchers developed mouse models of human acute myeloid leukemia (AML) that accurately predict response to chemotherapy and identify key genes promoting resistance or sensitivity. These models provide valuable insights into AML's genetic heterogeneity and may help redesign therapy for maximum benefit.
Researchers at UW-Madison have developed a new method to generate induced human pluripotent stem cells (iPS) without using viral vectors or exotic genes. This breakthrough removes key safety concerns and genetic artifacts that could compromise therapeutic safety or skew research results.
Researchers discovered that GDNF delivery to specific brain areas can cause unwanted weight loss in Parkinson patients. The finding suggests that gene therapy techniques in the brain potentially could control obesity and highlights the importance of placing therapy in the correct region.
Researchers at Vanderbilt University School of Medicine have discovered that inhibiting an enzyme called 11-beta-HSD2 blocks COX-2 activity in human and mouse colorectal tumor cells, potentially providing a new approach to preventing colorectal cancer. This finding is significant because long-term inhibition of 11-beta-HSD2 did not cau...
Researchers at Caltech found that Caenorhabditis elegans worms respond rapidly to better conditions and develop optimistic genetic strategies. The worms' quick response is controlled by a vital cellular protein called RNA Polymerase II, which accumulates on genes involved in growth and energy metabolism.
A team of researchers, led by Dr. Robert Koenekoop, has discovered a new gene associated with Leber congenital amaurosis and retinitis pigmentosa, two devastating forms of childhood blindness. The SPATA7 gene identifies a crucial metabolic disease pathway and opens a new avenue for potential genetic therapies.
Scientists developed a method to measure the transport of therapeutic genes through the body using magnetic nanoparticles. The technology significantly increases gene transfer efficiency compared to non-magnetic methods, with potential for targeted cell delivery.
A new study found that gene therapy can be developed as a potentially effective treatment for HIV, reducing viral load and preserving the immune system. The technique involves delivering genes to cells to prevent viral replication, with promising results in a clinical trial involving 74 HIV-positive adults.
A clinical trial is underway to test if genetics can improve warfarin prescription by optimizing each patient's dose. Using genetic information, doctors may be able to better determine optimal warfarin doses, minimizing the risk of excessive bleeding or clotting in patients at the extremes of the dosing range.
Researchers have discovered genetic variants that can help personalize treatment for infant osteosarcoma, leading to improved survival rates and reduced toxicity. The study, which analyzed data from 100 patients, found that specific genetic variants are associated with greater methotrexate toxicity and better therapeutic effects.
Researchers at Virginia Commonwealth University identified a gene, AEG-1, that plays a key role in regulating liver cancer progression. The study found that AEG-1 expression increases as the tumor becomes more aggressive and is involved in invasion, metastasis, resistance to chemotherapy, and other processes.
Researchers at Rice University have created a precise image of a virus' protective coat, containing 5 million atoms. The image provides the clearest picture yet of the viruses' genome-encasing shell called a 'capsid', which could lead to new approaches for antiviral therapies and gene delivery.
Scientists have turned a benign adeno-associated virus into a highly infectious form that can effectively deliver a gene to lung cells, potentially curing cystic fibrosis. The new strain was created by forcing evolution to increase infectivity several hundred-fold, overcoming previous limitations in gene therapy.
Researchers have identified two common gene variants associated with increased blood pressure levels, which may lead to new therapies. The study, published in Nature Genetics, found that these variants affect proteins involved in cardiovascular response to stress, influencing blood pressure levels.
Scientists at Scripps Research Institute have found that anti-oxidants in the diet can help preserve function by adding them to their diet. They also developed a novel gene therapy technique to offer an alternative option for arresting vision loss from eye diseases.
Researchers have demonstrated that gene therapy can reduce pain and swelling in patients with advanced rheumatoid arthritis. The therapy involves implanting a normal gene to compensate for a defective gene, resulting in lower amounts of disease-related proteins and reduced symptoms.