Articles tagged with Gene Therapy
Researchers have discovered DNA hotspots associated with psoriasis, an autoimmune disease characterized by skin patches and joint pain. The study identified four novel genes linked to the condition, providing potential targets for new treatments.
Researchers have discovered a novel way in which the Myc oncogene accelerates cancer by directly affecting protein production, suggesting a new strategy to halt cancer's progression. Drugs like Rapamycin may help treat cancers where Myc is overactive.
Researchers have identified a new subtype of childhood T-lymphoblastic leukemia, also known as ETP-ALL, which is characterized by poor response to standard intensive chemotherapy. The disease has a dire prognosis, with high rates of remission failure and relapse compared to typical T-ALL patients.
A new nasal spray vaccine has shown promising results in protecting against botulism, a deadly bacterium that can cause paralysis and death. The vaccine uses a gene therapy approach to prime the immune system against a key toxin, offering a safer alternative to existing vaccines.
Researchers have discovered a new therapeutic target that improves deteriorating skeletal muscle tissue caused by SMA. The therapy enhances muscle strength, improves gross motor skills, and increases the lifespan in a SMA model. By inhibiting myostatin, a protein that limits muscle growth, the treatment reduces symptoms of SMA.
Researchers at Thomas Jefferson University's Center for Translational Medicine successfully reversed heart damage in rats with heart failure using gene therapy. The treatment, which involves administering a peptide called βARKct, inhibited the activation of a kinase that contributes to heart failure deterioration.
Researchers at Saint Louis University discovered genetic patterns that can predict which patients are likely to respond to hepatitis C treatment. The study identified 'covariance networks' in the genes of patients who responded and resisted therapy, revealing a complex web of amino acid interactions.
A genetic variation affecting the CYP2C19 enzyme makes anti-clotting drug clopidogrel less effective in young heart attack patients, increasing risk of death, another heart attack, or stent thrombosis. Carriers of the gene variation are four times more likely to experience cardiovascular events than non-carriers.
A team of researchers from VCU identified a new anti-tumor gene called SARI, which can inhibit the growth and survival of tumor cells by interfering with cancer cell molecules. The discovery highlights a previously unrecognized molecular pathway underlying the anti-tumor action of interferon.
Researchers have identified a possible new therapeutic target for pancreatic cancer, the SINA gene, which regulates cell growth and oncogenesis. Inhibiting this gene's function may lead to novel anti-cancer therapies.
Researchers at the University of Michigan have successfully treated periodontal disease using gene therapy, showing over 60-80% sparing of periodontal tissues. The approach delivers an inactivated virus to produce more of a naturally-produced molecule that soaks up excessive levels of tumor necrosis factor.
Smokers carrying a specific gene variant are more susceptible to concentration problems and cognitive deficits during abstinence, increasing the risk of relapse. The study paves the way for novel medications to treat nicotine addiction.
A Canadian and American research group has been awarded a five-year grant to develop gene therapies for human degenerative retinal diseases, including Leber Congenital Amaurosis, Stargardt macular dystrophy, and retinitis pigmentosa. The team will use innovative approaches to partially restore vision in patients.
Researchers at St. Jude Children's Research Hospital have developed a gene therapy that alleviates sickle cell disease pathology by introducing a corrective gene into mouse blood cells. The treated mice showed essentially no difference from normal mice, with improved red blood cell production and organ function.
Researchers have developed a new approach to modulate gene expression using miRNA natural gene repressors for therapeutic purposes, effectively treating cancer in mice. Additionally, the study has provided insight into the molecular mechanisms controlling epithelial fluid and HCO3– secretion, which may help understand cystic fibrosis.
Scientists have developed a new approach to harness the power of microRNAs (miRNAs) as natural gene repressors for therapeutic purposes. By engineering mouse bone marrow cells to express genes only when miR-181a is downregulated, they were able to create immune cells that could target and destroy cancer cells.
Researchers discovered a greater level of genetic complexity for diseases than thought, focusing on iminoglycinuria and hyperglycinuria disorders. The study found that up to four different pumps in the kidney determine the disorder's occurrence, suggesting a need for integrated complex genetic information.
A study published in Nature Genetics has identified three chromosome segments where common genetic variations can create significant risk for ruptured aneurysms, leading to strokes. The findings could lead to new screening tests and therapies to strengthen blood vessels in the brain.
Researchers at University of Cincinnati discovered a gene that helps control stomach acid production, which could lead to new therapies for overproduction. The study found that eliminating the Slc26a9 transporter gene stops acid secretion in the stomach.
Researchers have found an effective way to deliver gene therapy, targeting both skeletal and cardiac muscle, with the potential to treat Duchenne muscular dystrophy. The therapy could be beneficial for patients by correcting electrocardiogram abnormalities in their hearts.
Researchers developed a simple blood test that measures inflammatory gene expression to evaluate cystic fibrosis patients' response to therapy. The test identified genes CD36, CD64, and ADAM9 as accurate biomarkers for therapeutic response.
Researchers found that BRAF mutations are associated with resistance to anti-EGFR therapy in colorectal cancer patients. The study suggests that combination therapy blocking EGFR and BRAF may be a useful approach to increase patient benefits.
Researchers create customizable biological computer using synthetic RNA that can detect tumor cells and respond to biochemical signals. The device has potential medical applications, including targeted gene therapies and cancer treatment.
Scientists at the Institut de recherches cliniques de Montreal have discovered a novel mechanism regulating neural stem cell development in the retina. The Ikaros gene plays a crucial role in conferring early temporal competence to retinal progenitor cells, enabling them to generate specific cell types at different stages of development.
Researchers found that inactivating key genes involved in 'fat-burning' can increase energy expenditure and lower diet-induced obesity. By forcing the body to use less efficient methods, mice became resistant to obesity despite consuming a high-fat diet.
A new study found that patients with specific genetic variations lose more weight when taking the weight loss drug sibutramine and undergoing behavioral therapy. The research suggests gene pairs can predict treatment effectiveness, which could help personalize medicine for obesity treatment.
A study found three genes associated with an increased risk of gout, enabling the development of a genetic risk score that can predict those at highest risk. This score could facilitate early and targeted treatments, improving outcomes for individuals affected by gout.
Researchers at Massachusetts General Hospital have discovered a new approach to gene therapy that uses normal brain tissue to create a 'zone of resistance' against brain tumor growth. By delivering a cancer-fighting gene to the surrounding tissue, tumors can be suppressed and eventually eliminated.
A new study demonstrates that transferring human insulin genes to the gastrointestinal tract using a non-viral vector called chitosan nanoparticles can successfully treat type 1 diabetes in rats. The study found that this method is safe and efficient, without any wound or infection.
Researchers discovered a genetic connection between neurons and immune response cells in roundworms, showing that specific neurons can regulate immune responses. This finding could lead to new approaches to boost innate immunity against different pathogens.
Researchers at the University of Granada have identified the genes responsible for corneal edema-induced blindness. The study revealed that altered ion concentration in endothelial cells leads to barrier dysfunction and oedema. New treatments, including gene therapy, may soon be available to address this condition.
A safety study on gene therapy for a rare form of blindness has shown promising results, with three patients regaining some vision. The treatment involves delivering a gene called RPE65 to the retina using an adeno-associated virus, and has been found to be fully safe with no adverse effects beyond routine postsurgical soreness.
A large-scale study has identified a range of genetic mutations and signaling pathways disrupted in glioblastoma, the most common adult brain cancer. The findings validate the potential of unbiased genome analysis to lead to paradigm-shifting discoveries and identify new targets for treatment.
The Cancer Genome Atlas identifies new genetic mutations, including NF1, ERBB2, and PIK3R1, and core pathways disrupted in glioblastoma. The study provides an unprecedented overview of the genomic landscape of this deadly cancer.
Researchers have developed a gene therapy that prevents blindness in an animal model of mitochondrial dysfunction. The treatment, which involves allotropic expression of mitochondrial genes, restored normal function to human fibroblasts with mutated mtDNA, effectively rescuing animals from impending blindness.
A new study suggests that short interfering RNAs (siRNAs) may exhibit non-specific therapeutic effects related to immune stimulation. The researchers used a mouse model of influenza and demonstrated that the anti-viral activity of siRNA is mainly due to non-specific stimulation of the immune system.
A study has identified a new gene that may play a key role in the development of pediatric-onset inflammatory bowel disease. The gene, TNFRSF6B, is involved in regulating inflammation and its activity has been linked to the severity of the disease.
A new study found that an experimental therapy for age-related macular degeneration may have adverse effects on patients with a certain genetic mutation. The therapy uses RNA interference to silence disease-causing genes, but this approach may not be suitable for everyone due to individual genetic differences.
A study has identified the first gene associated with severe dry macular degeneration, a condition that can lead to blindness. The researchers found that individuals with a specific genetic variant may be at risk if treated with an experimental therapy called RNA interference.
Researchers have discovered a genetic mutation that offers protection against age-related macular degeneration (AMD), a leading cause of blindness in adults over 50. The study identifies a functional link between toll-like receptor 3 (TLR3) and geographic atrophy, a type of dry AMD.
University of Alabama at Birmingham researchers have developed a gene therapy virus to target ovarian cancer cells. The adenovirus-based approach has shown anti-tumor effects that appear safe for most patients, according to Drs. David T. Curiel and Ronald Alvarez.
Researchers developed PTC124 to bypass premature 'stop' signals in CFTR genes, allowing the protein-making machinery to function normally. The phase II trial showed improvements in chloride ion transport and lung respiratory function, offering a potential treatment for cystic fibrosis.
A novel mutation in the nephronophthisis 4 (NPHP4) gene has been identified as a potential cause of cone-rod dystrophy in standard wire-haired dachshunds. This discovery raises hopes for developing treatments for humans with similar eye disorders.
Researchers explore new treatments for psoriasis, revealing a potential therapeutic option in AEB071, while cautioning further clinical trials are needed. Additionally, studies uncover the reasons behind gene therapy complications in SCID patients, shedding light on insertional oncogenesis and acquired somatic mutations.
A multi-institutional team has developed a powerful tool for genomic research and medicine, generating synthetic enzymes that can target specific DNA sequences for inactivation or repair. This new method, called OPEN, enables customized zinc-finger nucleases with high efficiency in many cell types.
A team of researchers has developed a powerful tool to induce specific genomic modifications in many types of cells, including plants and humans. The new method, known as OPEN, enables academic researchers to rapidly create high-quality enzymes that can target and manipulate DNA sequences for repair or inactivation.
A study of Middle Eastern families with a high incidence of autism identified six new genes involved in the brain's ability to form new connections. The findings suggest that autism may stem from disruptions in experience-dependent learning, which is essential for learning and memory.
Researchers identified six genes associated with autism by analyzing large Middle Eastern families where both parents shared a recent ancestor. The study suggests that these genes affect learning and memory, and may be treatable through gene therapy or enriched learning environments.
Scientists at UGA create novel synthetic gene vectors that package DNA into nanostructures for efficient gene delivery without triggering immune responses. The study has implications for cancer treatment and vaccine development.
A small study of 18 patients with severe Marfan syndrome found that losartan slowed aortic enlargement by nearly half, offering a promising new treatment option. The study's findings have led to the approval of a large-scale clinical trial for losartan in Marfan syndrome patients.
The Indiana University School of Medicine will be home to the nation's sole National Gene Vector Biorepository and Coordinating Center for gene therapy research. The center will help researchers share information and substances to promote discoveries and patient safety.
Researchers developed a therapeutic system targeting HIF-1 activated cancer cells, showing success in laboratory tests. The system uses a reporter gene to detect and treat hypoxic cancer cells that are resistant to traditional treatments.
A new gene therapy has helped pet dogs with cancer live longer by increasing muscle strength and correcting common complications. The treatment has been shown to improve quality of life and survival rates, with 54% of dogs responding to the therapy and surviving 84% longer.
Research by NARSAD scientists provides clues on how genes impair attention, memory, and perception in psychiatric illnesses like schizophrenia, bipolar disorder, and depression. The study focuses on specific genes and family traits that contribute to the susceptibility and pathology of these diseases.
Researchers identified genetic variants that influence treatment outcome for smoking cessation treatments, including nicotine replacement therapy (NRT) and bupropion. These variants may help tailor treatment to individual smokers, increasing the likelihood of success.
Researchers at the Burnham Institute have demonstrated successful enzyme replacement therapy to prevent hypophosphatasia in mice, paving the way for future clinical trials. The treatment maintains healthy bone mineral density and preserves skeletal structures, offering new hope for patients with this genetic disorder.
Researchers have developed artificial viruses that can transport genes and drugs into cancer cells, eliminating immune responses and potential side effects. The viruses are self-assembled using a ribbon-like protein structure, allowing for precise control over their size and shape.
A University of Rochester team has identified approximately 100 genes that cooperate to cause malignant cell transformation, providing a potential roadmap for next-generation cancer therapies. These genes, known as CRGs, encode proteins that regulate cell signaling and metabolism.
New data reveals genetic variation in AKT1 affects cognition and grey-matter volume in the frontostriatal region, a brain area dysfunctional in individuals with schizophrenia. This association may increase the risk of developing the condition.
Researchers investigated using gene therapy to inhibit NHE1 in human gastric carcinoma cells, leading to apoptosis of tumor cells. The study found that inhibition of NHE1 reduced malignant phenotypes and restrained tumor cell survival.