Articles tagged with Gene Therapy
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.
Researchers at Thomas Jefferson University have demonstrated the long-term therapeutic effectiveness and safety of S100A1 gene therapy in a large animal model of heart failure. The therapy works by restoring normal levels of the calcium-sensing protein, preventing and reversing cardiac dysfunction.
Researchers used gene transfer to deliver a therapeutic gene that produces protein apoA-1, which removes harmful cholesterol from atherosclerotic lesions. This approach protected against atherosclerosis in rabbit studies, with minimal and stable disease measurements over 48 weeks after a single dose of gene therapy.
A team of researchers led by UCSF and the Vaccine and Gene Therapy Institute aim to eradicate HIV from the body using a combination regimen. They will define HIV's reservoir, understand its creation and maintenance, and test potential treatments.
Researchers have developed a novel gene therapy system using nanoparticles that can be freeze-dried and stored for up to three months. The technology shows promise in treating brain cancer with high efficacy and minimal risk of complications, offering a potential alternative to traditional therapies.
Scientists at Fox Chase Cancer Center have identified a novel approach to develop targeted cancer therapies by reversing silenced genes through demethylation. The study suggests that specific proteins, such as thymine DNA glycosylase, can actively remove methyl groups from DNA.
Gene therapy using stem cells holds great potential for treating a range of diseases, thanks to advances in gene marking techniques, PCR sequencing, and chromatin insulators. The success of these methods could lead to improved patient survival rates.
A team of researchers has discovered a naturally occurring disease in monkeys that shares similarities with multiple sclerosis, providing new insights into the cause of the disease. The discovery is associated with a herpes virus and could lead to significant advancements in MS research and treatment.
Researchers at Mount Sinai School of Medicine developed a gene therapy called SERCA2a that stabilized or improved cardiac function in people with severe heart failure. Patients receiving the high-dose therapy experienced substantial clinical benefit and significantly reduced cardiovascular hospitalizations.
A new study found that a specific gene variant, PNPLA3 rs738409, is linked to an increased risk of developing fatty liver and faster fibrosis progression in people with chronic hepatitis C. The study suggests this genetic predictor may also be used as a potential therapeutic target for treating the condition.
New tools and methods enable targeted gene delivery to cells in the central nervous system, offering hope for new treatments. Novel strategies include using stem cells and siRNAs to silence genes involved in neurological diseases.
Researchers used genome editing to treat hemophilia in mice by precisely targeting and repairing mutated DNA. The treatment, which uses zinc finger nucleases, showed clinically meaningful results with no toxic effects or complications.
Researchers at the University of Pennsylvania have developed a new gene vector, AAV8, that delivers genes to photoreceptor cells more efficiently and safely than a previously used vector, AAV2. The study demonstrates potential for treating eye diseases such as retinitis pigmentosa.
Research suggests that many hereditary conditions are linked to errors in pre-mRNA splicing, which can be addressed through new therapies. A computer analysis predicts that 22 percent of genetic mutations may affect splicing, implying hundreds of hereditary diseases could be linked to this issue.
An experimental gene therapy has reversed type 1 diabetes in mice with a nearly 80 percent success rate, reversing autoimmune destruction of insulin-producing beta cells. The treatment uses neurogenin3 and betacellulin to stimulate new islet growth and inhibits immune system activity.
Research at the University of Texas MD Anderson Cancer Center found that customizing targeted therapies based on a patient's specific gene mutations can lead to higher response rates, survival, and failure-free survival compared to non-matched patients. This approach has shown promise in treating solid tumors with gene aberrations.
A gene discovery for migraine may lead to the development of a preventive pill that blocks the brain's pain channel. The discovery is significant as it opens understanding of the pain pathways that trigger migraine pain, sparking excitement among researchers and the pharmaceutical industry.
Researchers found loss of PTEN and mutations in MEK2, CDK4, and CDKN2A to be associated with patient response to GSK436. This study provides new insights into personalized approaches to melanoma therapy.
A study found that a specific gene change can predict which brain cancer patients respond well to treatment, allowing for personalized therapy. The gene MGMT methylation status was associated with improved overall and progression-free survival in patients treated with radiation and chemotherapy.
A phase 3 trial of a novel gene therapy failed to reduce amputations or deaths in patients with critical limb ischemia. The study highlights the need for effective treatments for this condition, which affects millions worldwide and results in significant healthcare costs.
Neurologix presents 1-year data from its Phase 2 study of NLX-P101, a gene therapy for Parkinson's disease, showing sustained improvements in motor symptoms. Patients treated with NLX-P101 experienced highly clinically meaningful symptom improvements, with a mean 37% improvement after one year.
A National Institutes of Health grant has been awarded to Nationwide Children's Hospital to develop a gene therapy for MPS IIIB, a fatal lysosomal storage disease. The therapy aims to deliver needed genes directly to the central nervous system by bypassing the blood-brain barrier.
Researchers have successfully isolated individual charged particles like DNA molecules using a method called Paul trapping, which could lead to personalized diagnosis and treatment. The device is small, inexpensive, and can be used on a single chip, allowing for rapid testing of patient samples in offices.
Researchers identified a new probiotic bacteria-derived soluble protein that can protect intestinal cells from inflammation and injury in mice models of colitis. The protein's delivery to the colon provided therapeutic protection against ulcerative intestinal inflammatory disorders.
Researchers at Salk Institute successfully edit a diseased gene in patient-specific induced pluripotent stem cells and adult stem cells using a virus-based approach. The method provides an efficient and safe tool for cell engineering, opening the way for gene editing-based stem cell therapies suitable for clinical applications.
Advances in gene therapy success depend on the development of viral delivery vectors. Researchers have made progress in refining AAV-based vectors, improving their production and use. This has enabled clinical proof of concept and paved the way for commercialization.
Researchers combine Sabutoclax with viral gene therapy to prevent tumor growth in prostate-cancer-prone mice, offering a novel approach to treating advanced prostate cancer. The study's findings suggest that this combination therapy could be effective in other cancers and pave the way for personalized medicine.
Recent research highlights exciting developments in ocular gene therapy, including potential treatments for age-related macular degeneration and retinal disease. Gene therapy strategies have shown promise in reducing cell death and slowing retinal degeneration.
Researchers at Tufts University School of Medicine developed a gene therapy approach that significantly reduces uncontrolled blood vessel growth and cell death typical of AMD, slowing its progression. The treatment uses CD59 to block the formation of 'membrane attack complex' in cell membranes.
Researchers developed an effective gene therapy technique using induced pluripotent stem cells to correct chronic granulomatous disease in cell culture. The corrected neutrophils produced normal levels of hydrogen peroxide, potentially offering a cure for the rare disorder.
Researchers at University of Michigan have developed a novel gene therapy, NP2, that shows promise in providing substantial pain relief for patients with unrelenting cancer-induced pain. The study demonstrates the safety and efficacy of NP2 in reducing pain by up to 80% over four weeks.
Researchers have determined that correcting a genetic defect does not substantially increase the number of potentially cancer-causing mutations in induced pluripotent stem cells. This breakthrough suggests that human-induced pluripotent stem cells altered to correct a genetic defect may be cultured into subsequent generations of cells ...
A study found that young adult patients with hypertrophic cardiomyopathy survived cardiac arrest after receiving therapeutic hypothermia. The treatment was successful despite marked left ventricular wall thickness and other risk factors.
Researchers successfully reprogrammed diseased human hepatocytes into induced pluripotent stem cells, offering a potentially unlimited source for liver disease treatment. This breakthrough could enable the generation of genetically corrected liver cells via auto-transplantation, avoiding liver transplants and immunosuppression.
A double-blind gene therapy trial has demonstrated safety and significant improvement in motor function for patients with Parkinson's disease. The study found that injecting the glutamic acid decarboxylase (GAD) gene directly into the brain improved motor scores by 23.1% compared to a placebo group.
A gene therapy called NLX-P101 significantly reduces movement impairment in Parkinson's patients, with half achieving dramatic symptom improvements. The treatment introduces a gene to normalize chemical signaling, correcting dysfunction in brain circuitry responsible for coordinating movement.
A new study found that gene therapy NLX-P101 improved motor performance and control of Parkinsonism symptoms in half of patients, offering hope for the treatment of neurodegenerative disorders. The therapy has been safe and well-tolerated over a six-month period.
A double-blind trial demonstrated a 23% improvement in motor symptoms among patients receiving gene therapy for advanced Parkinson's disease, compared to a 12% improvement in those receiving sham surgery. The study suggests a potential breakthrough in treating this progressive movement disorder.
A study at Cold Spring Harbor Laboratory has identified a therapeutic target for liver cancer using a monoclonal antibody that blocks the activity of the FGF19 gene. This approach shows promise in treating patients with FGF19 amplification, which can be used as a biomarker to predict treatment effectiveness.