Research on treating disease by editing or delivering genes. Covers sickle-cell gene therapy, gene-editing nanoparticles, mRNA delivery, and blood-cell-inspired carriers.
Researchers at VIB develop efficient and safe gene delivery approach based on non-viral genetic elements called transposons, overcoming limitations of viral vectors. This technology may simplify gene therapy, improve safety and reduce costs, treating cancer and genetic disorders.
Researchers at Yale University have developed a novel approach to deliver small interfering RNA (siRNA) molecules using biodegradable nanoparticles, achieving sustained release and effective knockdown of gene activity. This breakthrough holds promise for the treatment of sexually transmitted diseases (STDs), particularly HPV and HIV.
Scientists at the University of California, Berkeley and Los Alamos National Laboratory have developed a comprehensive numerical study of gene therapy. The research sheds light on the key factors that determine the success of dendrimers as gene delivery vehicles, including the charges of the dendrimers and their target cell membranes.
Research found that women with tumors carrying TOP2A gene alterations had better outcomes when treated with anthracycline-based regimens, similar to those seen in HER2-positive tumors. The study suggests that TOP2A gene alteration is associated with increased benefit from anthracycline therapy.
The 157th ASA Meeting explores various disciplines and unique sounds. Wimps hear dangerous noises differently due to physical fitness, while fish living in darkness have exceptionally sensitive ears. Researchers also study medical devices using sound to treat disease, a long-gone whale's comeback, and cowards' evolution.
Researchers at Fox Chase Cancer Center identified a genetic pattern that may predict how patients respond to imatinib mesylate, and found genes that could be suppressed to enhance therapy effectiveness. The study's findings suggest potential new strategies for improving treatment outcomes in gastrointestinal stromal tumor patients.
Researchers at M. D. Anderson Cancer Center discovered genetic variations in the inflammation pathway that reduce bladder cancer recurrence and increase survival. Patients with risk-reducing genotypes had a 84% lower likelihood of disease recurrence after BCG treatment.
Researchers successfully transferred a gene to produce a protein necessary for healthy muscle fiber growth, increasing muscle-fiber size and producing the needed protein for at least six months. The findings demonstrate the feasibility of gene therapy for treating limb-girdle muscular dystrophy and other muscle disorders.
Researchers at the University of Michigan have developed a gene therapy approach that safely regenerates gum tissue. The method uses a localized application of adenovirus to deliver genes directly to the affected area, reducing the risk of systemic reactions.
Researchers suggest that autism symptoms may be reversible due to dysregulation of the locus coeruleus, a bundle of neurons processing sensory signals. The new theory proposes that fever can stimulate this system, potentially restoring its normal function.
Researchers developed mouse models of human acute myeloid leukemia (AML) that accurately predict response to chemotherapy and identify key genes promoting resistance or sensitivity. These models provide valuable insights into AML's genetic heterogeneity and may help redesign therapy for maximum benefit.
Researchers at UW-Madison have developed a new method to generate induced human pluripotent stem cells (iPS) without using viral vectors or exotic genes. This breakthrough removes key safety concerns and genetic artifacts that could compromise therapeutic safety or skew research results.
Researchers discovered that GDNF delivery to specific brain areas can cause unwanted weight loss in Parkinson patients. The finding suggests that gene therapy techniques in the brain potentially could control obesity and highlights the importance of placing therapy in the correct region.
Researchers at Vanderbilt University School of Medicine have discovered that inhibiting an enzyme called 11-beta-HSD2 blocks COX-2 activity in human and mouse colorectal tumor cells, potentially providing a new approach to preventing colorectal cancer. This finding is significant because long-term inhibition of 11-beta-HSD2 did not cau...
Researchers at Caltech found that Caenorhabditis elegans worms respond rapidly to better conditions and develop optimistic genetic strategies. The worms' quick response is controlled by a vital cellular protein called RNA Polymerase II, which accumulates on genes involved in growth and energy metabolism.
Scientists developed a method to measure the transport of therapeutic genes through the body using magnetic nanoparticles. The technology significantly increases gene transfer efficiency compared to non-magnetic methods, with potential for targeted cell delivery.
A team of researchers, led by Dr. Robert Koenekoop, has discovered a new gene associated with Leber congenital amaurosis and retinitis pigmentosa, two devastating forms of childhood blindness. The SPATA7 gene identifies a crucial metabolic disease pathway and opens a new avenue for potential genetic therapies.
A new study found that gene therapy can be developed as a potentially effective treatment for HIV, reducing viral load and preserving the immune system. The technique involves delivering genes to cells to prevent viral replication, with promising results in a clinical trial involving 74 HIV-positive adults.
A clinical trial is underway to test if genetics can improve warfarin prescription by optimizing each patient's dose. Using genetic information, doctors may be able to better determine optimal warfarin doses, minimizing the risk of excessive bleeding or clotting in patients at the extremes of the dosing range.
Researchers have discovered genetic variants that can help personalize treatment for infant osteosarcoma, leading to improved survival rates and reduced toxicity. The study, which analyzed data from 100 patients, found that specific genetic variants are associated with greater methotrexate toxicity and better therapeutic effects.
Researchers at Virginia Commonwealth University identified a gene, AEG-1, that plays a key role in regulating liver cancer progression. The study found that AEG-1 expression increases as the tumor becomes more aggressive and is involved in invasion, metastasis, resistance to chemotherapy, and other processes.
Researchers at Rice University have created a precise image of a virus' protective coat, containing 5 million atoms. The image provides the clearest picture yet of the viruses' genome-encasing shell called a 'capsid', which could lead to new approaches for antiviral therapies and gene delivery.
Scientists have turned a benign adeno-associated virus into a highly infectious form that can effectively deliver a gene to lung cells, potentially curing cystic fibrosis. The new strain was created by forcing evolution to increase infectivity several hundred-fold, overcoming previous limitations in gene therapy.
Researchers have identified two common gene variants associated with increased blood pressure levels, which may lead to new therapies. The study, published in Nature Genetics, found that these variants affect proteins involved in cardiovascular response to stress, influencing blood pressure levels.
Scientists at Scripps Research Institute have found that anti-oxidants in the diet can help preserve function by adding them to their diet. They also developed a novel gene therapy technique to offer an alternative option for arresting vision loss from eye diseases.
Researchers have demonstrated that gene therapy can reduce pain and swelling in patients with advanced rheumatoid arthritis. The therapy involves implanting a normal gene to compensate for a defective gene, resulting in lower amounts of disease-related proteins and reduced symptoms.
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.
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.
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.
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 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.
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.
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.