Articles tagged with Gene Therapy
Researchers at Ohio State University Comprehensive Cancer Center discovered a small inherited change in DNA responsible for overactivating the BAALC gene, which is associated with poor treatment response in acute leukemia. The study found that high levels of RUNX1 protein are linked to high BAALC gene expression.
A recent clinical trial found that gene therapy can insert the correct ADA gene into patient bone marrow cells, leading to a good response. Defects in B cell tolerance are also corrected after gene therapy, supporting its use as an effective treatment option for ADA-deficient severe combined immunodeficiency patients.
Researchers developed a new method to identify vaccine targets for Streptococcus pneumoniae, the most common cause of bacterial meningitis. Gene therapy has shown promise in treating adenosine deaminase deficiency by correcting B cell tolerance problems and supporting treatment options for patients with severe combined immunodeficiency.
Experimental video game technology helps therapists monitor patients' arm rehabilitation after stroke, improving recovery outcomes. A UK-led gene therapy approach has shown promising results in treating hereditary blindness, with no adverse effects reported.
Taiwanese doctors use gene transfer to treat children with a rare neurological disease, restoring movement and improving cognitive development. The treatment has implications for Parkinson's disease and other neurodegenerative disorders.
Researchers found two specific gene variants associated with faster motor decline in Parkinson's patients. These variants can now help doctors predict disease progression, allowing for targeted therapies and faster results on efficacy of drugs.
Researchers found that injecting DNA nanoparticles increased expression of indoleamine 2,3 dioxygenase (IDO), an enzyme that calms the immune response, significantly reducing limb joint swelling and inflammation in rheumatoid arthritis. This suggests a potential therapeutic value for gene therapy in autoimmune diseases.
Researchers at CNIO successfully test first gene therapy to combat aging, extending mouse lifespan up to 24 percent and improving health. The therapy delivers a 'rejuvenating' effect using telomerase enzyme, repairing or delaying DNA damage.
A UNC-led team discovers that lung cancer molecular subtypes correlate with distinct genetic alterations and patient response to therapy. The study refines their previous report on three molecular subtypes of non-small cell lung cancer, suggesting new avenues for targeted treatment.
Scientists at NC State University developed a method to turn gene expression on and off using light-activated molecules. This technique enables precise control over gene function, potentially leading to targeted therapies for diseases like cancer.
Positive results from animal models and initial clinical trial results show promise for retinal gene therapy to treat inherited diseases. Researchers have developed efficient and safe viral delivery systems to introduce therapeutic genes into photoreceptor cells.
Researchers from the University of Pennsylvania report that genetically modified T cells remain healthy up to 11 years after initial therapy in a decade-long study of HIV patients. The approach provides a framework for gene therapy as a powerful weapon in treating HIV, cancer, and other diseases.
A geneticist has developed a technique to test the functional differences of human gene variants using yeast cells. This method identified 37% of alleles associated with vitamin B6 supplementation for treating homocystinuria, a metabolic disease causing blood clots and mental retardation.
Researchers at Bascom Palmer Eye Institute have developed a novel gene therapy to treat Leber Hereditary Optic Neuropathy (LHON), an inherited genetic defect that causes rapid vision loss. The treatment successfully replaces mutated genes with healthy ones, restoring visual function in experimental models.
Researchers found vitamin D can stimulate bone cells to remove calcium from bones, potentially leading to density loss and increased fracture risk. This discovery may explain conflicting results of clinical trials examining the effects of vitamin D supplements on preventing fractures in elderly patients.
Scientists at IRB Barcelona and BSC successfully extracted structural information from a triple DNA helix in gas phase, preserving its biological environment. This breakthrough could lead to the development of antigen therapy based on these DNA structures.
Researchers use groundbreaking gene sequencing technology to rapidly detect FLT3 mutations in AML patients who have relapsed on therapy. This discovery may help develop new therapies to treat AML, a type of leukemia characterized by rapid white blood cell growth.
Scientists have developed a new technique to determine which patients with homocystinuria are most likely to respond to vitamin B6 treatment based on their genotypes. The study correlates specific gene mutations with disease severity and may help physicians prescribe treatment based on genotype.
A preclinical study found that patients with KRAS gene mutations have a worse prognosis and do not respond well to targeted therapies. However, patients with KRAS-mutant tumors may respond well to treatment with antifolates, especially if the mutation is not amplified.
Cardiomyopathy is a deterioration of the heart muscle affecting its pumping ability. MDC-researchers identified RNA binding motif protein 20 (RBM20) as a gene regulating titin splicing, a process connected to the disease. Understanding this mechanism may lead to more efficient molecular diagnosis and therapies for cardiomyopathy.
Researchers have discovered gene variations that predispose cystic fibrosis patients to develop an intestinal blockage while still in the uterus. These variants involve genes responsible for ion transport in the lower end of the small intestine, increasing the risk of meconium ileus and serious health problems.
The University of Ottawa Heart Institute's bedside genetic test has successfully protected patients with a specific genetic variant from adverse events. The test uses a simple cheek swab and has been shown to be effective in reducing high on-treatment platelet reactivity, a marker for complications after stenting.
Researchers have designed a powerful gene therapy strategy to treat beta-thalassemia and sickle cell anemia by transferring a healthy beta-globin gene into diseased cells. The new technique has shown promising results, with increased production of normal hemoglobin in patients.
Researchers found that certain genetic mutations in acute myeloid leukemia patients predicted improved outcomes when treated with high-dose induction chemotherapy. Mutational profiling could help identify distinct subgroups of patients who may benefit from dose-intensified therapy.
A genetic pathway previously known for its role in embryonic development and cancer has been identified as a target for systemic sclerosis, or scleroderma, therapy. The finding reveals that the Wnt signaling pathway is abnormally activated in scleroderma patients, leading to fibrosis and tissue damage.
Researchers have discovered a dramatic improvement in life span and motor function in mice with infantile Batten disease when treated with gene therapy and bone marrow transplants. The combination therapy created a striking synergy, with mice living nearly 18.5 months, more than double the lifespan of untreated mice.
A groundbreaking gene therapy trial for cystic fibrosis will begin in March, involving 130 adults and children with the disease. The trial aims to assess whether repeated doses of gene therapy can improve symptoms and lung function in patients.
Researchers have identified genetic abnormalities that help doctors predict patient prognoses and guide treatment decisions for AML. The study shows that nearly two-thirds of patients can be categorized into clear prognostic groups, leading to improved treatment outcomes.
Researchers found gene mutations in DNMT3A and NPM1, as well as translocations, associated with improved patient outcomes when treated with higher doses of daunorubicin chemotherapy. This discovery could help physicians tailor treatment plans for individual patients.
A genetic signature has been identified for eosinophilic esophagitis (EoE), a disease causing painful food allergies, inflammation, and swallowing difficulties. The microRNA signature is reversible with steroid treatment, offering an opportunity for non-invasive diagnosis and precise analysis.
Researchers successfully deliver genes for PDGF-BB and VEGF to mice, restoring blood flow and repairing damaged tissue without toxic effects. The balanced delivery of these factors shows promise for treating atherosclerotic disease and preventing limb amputations.
Researchers have found that an epigenetic blockade affects people with Alzheimer's disease, leading to a blockade of genes involved in learning and memory. A treatment approach using gene therapy reduced HDAC2 levels, preventing the blockade and improving neuroplasticity.
A study in a mouse model of Down syndrome identifies increased expression of protein Dyrk1a as a promoter of acute megakaryoblastic leukemia, offering a candidate therapeutic target for treatment.
A computer model used by researchers at Stanford University found that new triple-therapies for genotype-1 hepatitis C are cost-effective for patients with advanced disease. The treatment options have more severe side effects and boost costs, but help these patients avoid costly cancers and liver transplants.
Researchers report growing success in combining therapeutic cancer vaccines with conventional chemotherapy, stimulating the immune system to destroy cancer cells. The first FDA-approved cancer vaccine is for metastatic prostate cancer, and several other strategies are being tested for various types of cancer.
Researchers successfully improved vision in three adult patients who previously received gene therapy in one eye, achieving better light sensitivity and navigating obstacles in dim light. No immune reactions occurred during the readministering treatment, and unexpected benefits were observed in brain responses.
Researchers found a gene, LTA4H, that influences inflammation to TB infection, predicting drug therapy effectiveness. This discovery suggests tailoring treatment based on patients' genetic sequences could improve patient outcomes.
Researchers found that a single 'Goldilocks' gene, LTA4H, regulates the immune response to TB. Variations in this gene lead to excessive or insufficient inflammation, which can affect treatment response. The study suggests personalized medicine could improve TB treatment outcomes.
Researchers have discovered new mechanisms by which RNA drugs can control gene activity, including binding to Argonaute proteins and other forms of non-coding RNAs. This breakthrough provides exciting new options for targeting emerging forms of non-coding RNAs and pre-mRNAs.
A new lung cancer assay measures gene activity in tumor tissue and provides a risk score to predict patient survival after surgery. The assay has been validated in two independent trials and shows promise in identifying patients at highest risk of mortality.
Gene therapy is poised to disrupt traditional treatment methods with its effectiveness in treating diseases such as hemophilia B. The technology has demonstrated feasibility and is expected to bring significant changes to the healthcare marketplace.
Researchers at Penn Vet & Scheie Eye Inst. successfully treated dogs with X-linked Retinitis Pigmentosa using gene therapy, correcting defects in the RPGR gene and restoring vision. The treatment targets both rod and cone cells, showing promise for treating other photoreceptor degenerations.
Researchers develop gene therapy to correct X-linked retinitis pigmentosa, a genetic defect causing peripheral and night vision loss. The technique replaces a malfunctioning gene with a normal one, supplying a protein for light-sensitive cells to function.
Researchers at the Gladstone Institutes have identified a key genetic mechanism linked to congenital heart disease, revealing the importance of epigenetics in fetal heart development. The study highlights the role of Ezh2 and Six1 genes in regulating healthy heart development, which can have profound health consequences later in life.
A University of Illinois study found that personalized prognostic tools and gene-based therapies can improve the survival and quality of life of glioblastoma patients. The researchers discovered new general and clinical-dependent gene profiles that can be used to predict patient outcomes and select targeted therapies.
Scientists have solved the three-dimensional structure of a newly discovered type of gene-targeting protein called TAL effector, which has a unique LEGO-like modular architecture. This discovery enables researchers to engineer the protein for targeted gene modification, genetic engineering, and corrective gene therapy.
Researchers distinguish cancer subtypes and provide a more complete understanding of Type 2 papillary renal cell carcinoma (PRCC2), an aggressive type of kidney cancer. They also identify genes involved in clear cell renal cell carcinoma (CCRCC) development and progression, highlighting PLK1 as a promising potential therapeutic target.
New research reveals that many bacteria try to fend off fluoride by throwing it out, and that the presence of this transport system indicates fluoride has antimicrobial properties. The discovery also highlights a genetic switch called riboswitches, which can be used to enhance fluoride's effects against bacteria.
Researchers used AAV vectors to increase NGF levels in the hearts of diabetic mice, preventing cardiac dysfunction and preserving blood flow. The study suggests NGF gene therapy may have tremendous therapeutic potential for treating diabetic cardiomyopathy.
A recent clinical trial in patients with hemophilia B showed that Factor IX gene therapy was able to convert severe hemophilia to moderate or mild disease. The treatment demonstrated a sustained therapeutic effect and eliminated or substantially reduced the need for standard protein replacement.
Researchers at St. Jude Children's Research Hospital and University College London have achieved early success with a gene therapy developed to treat hemophilia B. The treatment increased Factor IX levels in adults with the disorder, reducing the need for clotting factor injections to prevent bleeding episodes.
A Phase III clinical trial found that adding gemtuzumab ozogamicin to standard chemotherapy improved event-free and overall survival in newly-diagnosed acute myeloid leukemia patients aged 50-70. The treatment also reduced toxicity while providing therapeutic benefits.
Researchers at the Salk Institute have developed a new gene editing technique that uses patients' own cells to correct genetic mutations in the HBB gene, which causes sickle cell disease. The method repairs the beta-globin gene without introducing harmful genes into cells and appears to be more efficient than traditional techniques.
A new study finds that sewage treatment plants can be a significant source of antibiotic-resistant genes in waterways. Researchers detected three genes that make bacteria resistant to tetracycline antibiotics at a facility on Lake Superior, highlighting the potential for even high-tech plants to contribute to the problem.
Scientists have developed a new approach to gene therapy using site-specific recombinases from yeast and phages, allowing for precise genetic modifications. This technique has the potential to improve efficiency and effectiveness of experimental gene therapies while reducing side effects.
Researchers have successfully delivered replacement genes to patients with muscular dystrophy using a 'chimeric' virus. The study demonstrates the potential of customized gene therapy as a treatment option for this devastating disease.
Researchers have discovered a new way to build muscle by suppressing a natural inhibitor, resulting in mice and worms with super-strong muscles. This breakthrough could lead to treatments for age-related or genetics-related muscle degeneration, as well as applications for athletes and individuals with genetic muscular dystrophy.
Despite advancements in genetic profiling, breast cancer treatment remains non-personalized due to limited robust prognostic and predictive factors. Standard gene expression profiling has shown limitations in predicting chemotherapy benefit and prognosis for certain disease subsets.
Researchers are investigating a novel gene therapy approach using LG631 to improve tolerance and effectiveness of chemotherapy for glioblastoma, a devastating brain cancer. The study aims to prevent damage to bone marrow, enabling patients to receive higher doses with fewer side effects.
Researchers create super-strong mice and nematodes by reducing natural inhibitor function, leading to denser muscle fibers and increased energy delivery. This breakthrough could lead to treatments for age-related or genetically caused muscle degeneration.