Articles tagged with Gene Therapy
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 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 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 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.
Researchers have identified 15 new genetic regions associated with an increased risk of developing primary biliary cirrhosis. The study used genome data from 2,500 patients and 7,500 healthy individuals, tripling the number of known genetic regions to 22.
Researchers believe ADAM-12 gene could be important in fighting cancer, arthritis and cardiac hypertrophy. The gene's regulation pathway could lead to new treatment plans.
A new patented therapy has been developed by WSU researcher Dr. Karli Rosner, which uses genetic constructs to target cancer cells and induce apoptosis, eliminating the need for external medications. The treatment shows promise in treating various types of cancers, including melanoma, prostate, lung, and breast cancers.
Researchers identified AEG-1 as a strong predictor of progression-free survival in patients with EGFR-mutant non-small cell lung cancer treated with erlotinib. Patients with low AEG-1 expression had longer progression-free survival compared to those with high expression.
Researchers discovered a rare gene mutation strongly linked to schizophrenia, which could be a new target for effective treatment. The mutation affects the VIPR2 gene, leading to increased signaling in the Vasoactive Intestinal Peptide pathway.
A team of investigators has identified a new candidate gene for primary open angle glaucoma (POAG), the most common form of eye disorder. The gene, ADAMTS10, is involved in processing extracellular matrix and may play a role in regulating aqueous humor outflow.
A new gene test has identified GFPT1 as a crucial cause of Congenital Myasthenic Syndrome (CMS), a rare genetic condition affecting signal transmission between the brain and muscles. The test allows for earlier treatment with cholinesterase inhibitors, offering an effective therapy that can be taken through life.
Researchers have developed a new method for treating genetic diseases using modified mRNAs, which can be administered repeatedly without increasing the risk of cancer or severe immune reactions. In mouse models, this technique successfully restored lung function in mice with a congenital lung defect.
Researchers develop an innovative genetic strategy that transfers a bacterial gene into CD4+ T-cells, making them resistant to HIV infection. The MazF protein destroys gene transcripts, preventing protein synthesis and effectively blocking HIV replication.
Researchers found that adding caffeine to lentiviral production protocols increases virus production by 3-8 times. This simple and inexpensive strategy may significantly decrease the cost of lentiviral production for research and clinical uses.
Two studies found that ITPA variants protect against treatment-related anemia in HCV patients, enabling safer use of antiviral therapy and reducing the need for erythropoietin treatment. The variants also help select patients who may tolerate higher doses of ribavirin, increasing the likelihood of viral clearance.
Researchers at Duke University Medical Center discovered how nanoparticles from diesel exhaust damage lung airway cells, a finding that could lead to new treatments for people susceptible to airway disease. The study showed that the severity of injury depends on an individual's genetic make-up.
Dr. Sena-Esteves is investigating gene therapy techniques to replace the faulty gene causing Tay-Sachs Disease, a devastating neurological disorder. He's part of the Tay-Sachs Gene Therapy Consortium aiming to translate current results into a human clinical trial within three years.
Researchers have created a new type of nanoparticle called nanodisks, which can carry genetic material and enter cells quickly without losing its potency. These nanodisks offer a promising alternative to traditional gene therapy vectors, potentially improving therapeutic outcomes.
Researchers developed a gene therapy strategy using parthenogenetic embryonic stem cells, which can differentiate into multiple tissue types and potentially reduce rejection problems. The approach shows promise for treating autosomal dominant diseases like Beta Thalassemia, tuberous sclerosis, or Huntington's disease
Researchers from the University of Leicester and international partners have identified potential new therapeutic targets for Huntington's disease using cutting-edge genetic techniques. The study found that flavonoids can prevent cell death in yeast, suggesting a possible new treatment approach.
A recent study published in the New England Journal of Medicine found that losing the NFKBIA gene promotes glioblastoma multiforme growth and reduces survival. Restoring this gene may improve survival for certain patients with glioblastoma, as it inhibits tumor cell growth and increases sensitivity to chemotherapy.
A new, simplified method for producing large amounts of viral vector cassettes has been developed, overcoming previous limitations. This approach enables efficient packaging of single- or double-stranded AAV vectors and large AAV cassettes, paving the way for commercial production.
Researchers have developed a novel virus-based gene therapy for renal cell carcinoma, killing cancer cells both locally and distantly. The therapy combines Sorafenib with Ad.5/3-mda-7 adenovirus, which activates the MDA-7/IL-24 protein to stop tumor growth.
Scientists at Johns Hopkins and VCU have developed a nanoparticle-based gene 'ignition switch' to detect and treat cancer. The promoter-based system activates specific genes only in cancer cells, showing a 30-fold difference in identification.
A new $2.1 million NIH grant will help MU scientist Dongsheng Duan develop a treatment that prevents heart muscles from weakening due to Duchenne muscular dystrophy. Tests have shown that treating skeletal muscle alone can lead to complications, highlighting the need for comprehensive treatment targeting both skeletal and cardiac muscle.
Researchers at the University of Cincinnati found a potential genetic target, miR765, which can down-regulate protein phosphatase 1 inhibitor-1 and reduce cardiac muscle contractility. This micro-RNA may play a role in heart disease development by decreasing or deactivating proteins that help the heart function at full capacity.