Articles tagged with Gene Therapy
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.
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 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.
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 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.
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.
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.
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 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 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.
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.
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.
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 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.
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.
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.
Researchers at the University of Florida have developed a new version of the adeno-associated virus used in gene therapy that works more efficiently than current vectors. By replacing an amino acid on the surface of the virus, they were able to improve its ability to deliver genes into cells and reduce the risk of triggering an immune ...
Researchers presented findings on Maxi-K gene therapy, showing it to be safe and restoring erectile function in men with moderate to severe ED. The treatment has been shown to enhance sexual behavior, including increases in partial and full erections and a two-fold increase in erection duration.
Researchers have successfully treated late infantile neuronal ceroid lipofuscinosis (LINCL) with gene therapy, slowing disease progression in children. The treatment, which replaces the faulty CLN2 gene, demonstrated significant improvements over an 18-month period.
A clinical trial found gene therapy to be safe and effective at slowing disease progression in Batten disease, a rare genetic disorder. Neurological function was assessed using a rating scale throughout an 18-month follow-up period.
Researchers discovered glypican-3 loss induces overgrowth through Sonic Hedgehog, a growth factor stimulating cancer growth in rare disorder SGBS. This finding opens doors for novel treatments to inhibit overgrowth activity benefiting SGBS patients and GPC3-related cancer patients.
The American Society for Neural Therapy and Repair (ASNTR) has awarded neuroscientist Paul M. Carvey for his groundbreaking research on Parkinson's Disease, while recognizing patient advocate Joan Samuelson for her tireless advocacy efforts.
A new study by UC Davis researchers provides evidence that methods using human bone marrow-derived stem cells to deliver gene therapy do not cause the development of tumors or leukemia. The study tested the safety of gene transfer into bone marrow stem cells in over 600 mice, with none developing leukemia or solid tumors.
A genetic breakthrough explains dangerously high blood glucose levels in healthy individuals by identifying a DNA sequence that controls variability. This discovery holds out hope for 'personalized medicine' and may lead to customized treatment based on an individual's unique genetic structure.
Researchers have successfully used gene therapy to restore vision in three young adults with a rare form of congenital blindness. The study showed significant improvements in visual acuity and sensitivity to light, setting the stage for further studies.
Researchers developed a breakthrough in gene therapy for cancer using microscopic magnets to target tumours, increasing successful cell invasion. The 'magnetic targeting' method uses nanomagnets to attract cells carrying anti-cancer genes, effectively overcoming the problem of insufficient delivery.
New data shows that vectors derived from AAV5 can accommodate large genes, including those missing in a mouse model of recessive Stargardt disease. This breakthrough has implications for treating individuals with the disease, which is characterized by progressive loss of sight.
Cardiology researchers at The Children's Hospital of Philadelphia have developed a synthetic material that can bind to various gene therapy vectors, allowing controlled local release of therapeutic genes. In an animal study, the new formulation showed significant reduction in restenosis, a hazardous narrowing of blood vessels.
Researchers have discovered a new vector, AAV5, that can carry large genes linked to inherited blindness, paving the way for potential gene therapy treatments. This breakthrough enables the delivery of corrected faulty genes into cells where they are required.
Researchers have identified two potential biomarkers that could help doctors monitor the effectiveness of treatment with sunitinib or bevacizumab for kidney and non-small cell lung cancer. A study found that circulating endothelial progenitor cells (ccEPCs) increase in patients who respond to therapy, while those who do not respond hav...
Researchers have identified a crucial role for antibodies in protecting against nontyphoidal strains of Salmonella bacteria, which causes fatal infections in African children. Meanwhile, a study on HOXB4 gene therapy found that early precursors can cause leukemia in large animals, highlighting the need for extreme caution in human trials.
Researchers at St. Jude Children's Research Hospital are investigating two areas of molecular science that hold promise for improving the survival and quality of life of ALL patients, including adolescents and adults. Gene-expression profiling and pharmacogenetics may help identify targets for future therapy and reduce toxicity.
Researchers at Northwestern University discovered a counterintuitive approach to gene therapy that targets the removal of genes to restore cellular function. This method has implications for medical research and optimizing metabolic processes used in biofuel production.