Articles tagged with Gene Therapy
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.
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 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.
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.
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.
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.
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.
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 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.
A novel point-of-care genetic test has been shown to be clinically feasible and accurate, enabling rapid personalization of anti-platelet therapy for patients at risk. The study demonstrates that tailored treatment successfully protected all patients with the at-risk genetic variant from adverse events.
Researchers at Ohio State University Medical Center have developed a viral vector designed to deliver a gene into the eyes of people born with an inherited form of blindness. The trial aims to treat choroideremia, a disease affecting 100,000 worldwide, and holds promise for other genetic causes of blindness like retinitis pigmentosa.
Researchers have identified a new potential cancer drug targeting EPHA7, a tumor suppressor protein associated with follicular lymphoma. The discovery demonstrates the power of functional genomics in translating genetic insights into therapies.
Researchers have developed a new animal model to study Progeria and tested a gene therapy that significantly extended the lifespan of mice. The treatment, using 'vivo-morpholino' antisense oligonucleotide technology, reduced progerin production and improved various parameters related to the disease.
A Scripps Research scientist has been awarded a $500,000 grant to study the genetic mutations associated with Parkinson's disease. The researcher will investigate two genes, LRRK2 and SGK1, which have shown a link between their mutations and reduced risk of Parkinson's disease.
Researchers will combine neuroscience and engineering to repair spinal injuries and hearing loss using gene therapy, flexible electronics, optical imaging, and human-machine interfaces. The partnership aims to bring new treatments to the clinic and alleviate human suffering caused by neurological disabilities.
Researchers at Wayne State University developed a novel gene therapy approach to restore vision in people with age-related macular degeneration and retinitis pigmentosa. A license agreement has been signed with RetroSense Therapeutics, paving the way for Phase I clinical trials.
Scientists at Ohio State University have created a technique called nanochannel electroporation (NEP) that allows for precise injection of genes and proteins into individual cells. The method uses electrical pulses to deliver therapeutic agents, with potential applications in cancer diagnosis and treatment.
Researchers successfully used induced pluripotent stem (iPS) cells to treat a mouse model of a rare genetic liver disease. The study demonstrates the potential for iPS cells to be used in human gene therapy to counter pathological effects and promote liver regeneration.
A new study by Loyola University Chicago Stritch School of Medicine could lead to improved gene therapies for conditions such as heart disease and cancer. Researchers found that a virus used in vaccines can also be tailored to cause less of an immune response in gene therapy applications.
Researchers developed a novel bipartite gene therapy approach to temporarily preserve photoreceptors in a mouse model of retinitis pigmentosa. The treatment targets defective phosphodiesterase metabolism, reducing cGMP and Ca2+ levels, and showing promise for treating this genetic disorder.
A genetic variant in the GLCCI1 gene may explain why some people with asthma do not respond well to inhaled corticosteroids. Researchers found that individuals with two copies of the variant responded only one-third as well to steroid inhalers as those with regular genes.
A study on mice shows that reducing IRS2 signaling can slow the progression of Huntington disease. Meanwhile, research also finds that circulating glucose levels can modulate neural control of desire for high-calorie foods in humans. These findings provide potential therapeutic targets and new insights into the biology of obesity.
Researchers identified 16 new gene regions associated with blood pressure and confirmed 12 previously discovered ones, providing insights into the genetic mechanisms of hypertension. The study's findings could lead to new therapeutic drug development and improved treatment options for individuals with high blood pressure.
Scientists developed an electrically neutral matrix to hold DNA fragments, which spontaneously align and form ordered structures at high concentrations. This technology has potential applications beyond gene therapy, including the delivery of chemical drugs.
Researchers at Mount Sinai School of Medicine have identified SUMO1 as a potential therapeutic target for treating and preventing the progression of heart failure. Gene therapy with SUMO1 improved cardiac function in human and pig hearts, suggesting its critical role in pathogenesis.
A novel gene therapy approach combining with radiation therapy has been found to be safe and effective in treating glioblastoma multiforme, a deadly form of brain cancer. The treatment stimulates an immune response against the tumor, producing an 'immunogene therapy' effect.
Researchers at UNC School of Medicine have devised a gene therapy cocktail that can treat some inherited diseases caused by misfolded proteins. The approach uses an adeno-associated virus (AAV) vector to deliver two payloads simultaneously: one disables the mutant protein and another provides a new gene to replace its activity.
Researchers at National Physical Laboratory develop a model peptide sequence dubbed GeT that transports genetic material into human cells, overcoming poor cell membrane permeability. This approach holds promise for gene therapy in treating defective genes such as cancer.
A new study develops and tests genetically engineered spider silk for safe and efficient gene delivery, offering a promising alternative to viral vectors. The material successfully attaches to diseased cells and injects DNA without harming mice in lab studies.
Researchers found that individuals lacking TLR1 proteins are more susceptible to tuberculosis, leading to decreased immune function. The study suggests a potential approach for personalized therapy and prioritizing drug treatment in developing countries.
A study published in The Lancet reveals that targeted antisense therapy AVI-4658 can restore expression of a key protein lacking in patients with Duchenne muscular dystrophy. The treatment showed significant dose response, allowing boys to produce functional dystrophin protein up to 18% of normal levels.