Articles tagged with Gene Therapy
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.
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.
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 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.
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.
Researchers made a significant breakthrough in understanding the physics of translocation, showing that memory effects in polymeric molecules dominate their behavior. This discovery has major implications for drug delivery and gene therapy, as well as single-molecule characterization techniques.
Researchers have successfully treated mice with a gene responsible for making follistatin, a protein that inhibits myostatin, leading to long-term improvements in muscle mass and strength. The therapy has potential for older patients with Duchenne muscular dystrophy, who often lose treatment options once muscles degenerate.
Researchers identified a genetic variation in the VKORC1 gene that determines a patient's initial sensitivity to warfarin treatment. The study found that only variations of VKORC1 predict response within the first week of therapy, which could lead to better warfarin dosing and increased safety.
Researchers developed gene therapy approach that attracts and 'trains' immune system cells to destroy deadly brain cancer cells, promoting long-term immunity and restoring normal brain function. The therapy shows promise as a potential treatment for glioblastoma multiforme, the most common and deadly type of brain cancer.
Researchers at University College London have identified a combination of genes in Asian monkeys that protects against retroviruses, including HIV. By mimicking these genes in humans, scientists hope to develop a novel gene therapy treatment for HIV/AIDS.
Researchers at McGill University have discovered a genetic process that boosts an organism's natural anti-virus defences by knocking out genes repressing interferon production in mice. This breakthrough could lead to the development of new anti-viral therapies in humans, providing a potential solution to combat viral pandemics.
A recent MUHC study reveals that cystic fibrosis-related bone problems are an additional pathology stemming from the Cftr gene mutation, not a side effect of treatment. The study found that mice with the mutation have lower bone mineral density and mass than control mice, without pancreatic insufficiency or steroid treatment.
Researchers at Henry Ford Hospital are conducting a phase III clinical trial to determine the effectiveness of gene therapy in combination with radiation therapy for patients with intermediate risk prostate cancer. The trial aims to improve patient outcomes by delaying hormone therapy, which can have severe side effects.
Researchers have found that tattooing is a more effective way of delivering DNA vaccines than intramuscular injection, producing stronger humoral and cellular immune responses. This method could have a role in routine vaccination of cattle or delivering therapeutic vaccines to humans.
Researchers found that STAT3 has tumor-promoting and tumor-suppressing effects depending on the genetic profile of glioblastoma tumors. This discovery highlights the need for effective therapies tailored to individual glioblastoma tumors.
Scientists use gene therapy to breed goats producing therapeutic proteins in their milk, reducing drug development time and cost. This breakthrough increases drug availability for those who cannot afford treatments.
RNA interference represents an innovative strategy for silencing specific genes associated with disease processes, and a series of review articles will focus on its potential therapeutic applications. The technology has been approved for at least six clinical trials and is considered to be here to stay.
A series of commentaries in Human Gene Therapy explores key controversies surrounding patient recruitment and trial management in gene therapy trials. The authors discuss the challenges of patients comprehending lengthy and technical study descriptions and consent forms, and propose innovative strategies for improving informed consent.
Researchers at Mount Sinai School of Medicine have discovered a novel targeted gene therapy that simulates the pain-killing effect of opiate drugs, providing relief for more than three months after a single injection. The technique targets selectively the pain gate, avoiding unwanted side effects associated with opioid painkillers.
A landmark genetic study identified multiple genes linked to systemic lupus erythematosus (SLE), a debilitating autoimmune disease affecting joints, kidneys, heart, lungs, brain and blood. The findings will ultimately lead to new therapies and earlier diagnosis, with 13 strong candidates supported by data that are 99% accurate.
Researchers at Scripps Research Institute identified a nonsense mutation in the Coronin-1A gene that suppresses lupus development in mice. The study suggests that this mutation and other disease-suppressing genes may play a crucial role in modulating autoimmunity.
Researchers at UTMB have identified distinct subtypes of asthma using protein profiling, which could lead to tailored treatments. The study found four different asthma subtypes, including one associated with severe and treatment-resistant disease, offering new hope for more effective asthma therapies.
A new study published in Nature Genetics reveals that diet and lifestyle can significantly impact the effectiveness of certain drugs, including those used for cancer therapies. The research found that nutrient availability can either enhance or harm cell fitness, depending on the surrounding environment.
Researchers at Thomas Jefferson University have discovered that USP22 is crucial for controlling large-scale changes in gene expression in cancer cells. The study reveals that USP22 is necessary for the global changes in gene expression patterns that occur in these cells, making it a potential target for new anti-cancer drugs.
Researchers compared DNA of patients with reduced MS symptoms to those experiencing relapses, identifying key genetic markers associated with treatment response. The study's findings may help predict which treatments will work best for individual patients.
University of Florida researchers have successfully triggered insulin production in diabetic mice by injecting them with the naturally occurring protein Pdx1. The study found that daily injections of Pdx1 normalized blood glucose levels within two weeks, promoting regeneration of insulin-producing cells in the pancreas.
Researchers at the University of Rochester Medical Center have developed a new graft technique using freeze-dried tendon grafts loaded with gene therapy, showing significantly fewer adhesions than traditional autografts. The study demonstrates promising results in restoring range of motion and accelerating healing after hard-to-treat i...
China's health biotech industry boasts daring medical science innovation and stunning breakthroughs, including the world's first commercialized gene therapy product. However, firms face an uphill battle in attracting high-risk venture capital needed for innovative projects due to uncertain financial systems and export restrictions.
Researchers used rats bred to be heavy drinkers and administered an anti-Aldh2 antisense gene, reducing consumption by half for a month. The study suggests gene therapy could be a promising approach for treating alcoholism, particularly in individuals with genetic mutations protecting them from the risk of developing the condition.
Researchers identified brain regions that fire up more when people make impulsive decisions, a key finding linking thinking patterns to addiction. The study suggests raising dopamine levels may be an effective treatment for addiction, offering new approach to therapy and potential subtypes of alcoholics.
A research team has uncovered the molecular mechanism by which osteocrin controls bone growth, a discovery that may have important implications for people suffering from bone diseases affecting skeletal growth. Osteocrin was found to locally control the bone's supply of a hormone known as CNP, regulating bone growth.
Researchers have alleviated various abnormalities associated with fragile X syndrome by reducing the activity of a specific gene. The findings suggest that this approach may lead to treatment for the disorder, which affects brain structure and function, as well as causing mental retardation, epilepsy, and abnormal body growth.
A new study published by Mayo Clinic researchers found that Herceptin helps women with HER2+ type of breast cancer independent of whether they have extra copies of chromosome 17. The analysis showed significant benefits for patients with polysomy 17, including a disease-free survival rate of 89% after three years of treatment.
Researchers found that alcohol activates a stress-linked pathway in neurons to release key genes that can influence the health and activity of brain cells. The study sheds light on how this process occurs and may help scientists understand alcohol-linked disorders such as chronic alcoholism and fetal alcohol syndrome.
A study found that antidepressant non-responders with depression show a significantly better response to lithium augmentation if they carry at least one C-allele, suggesting a potential genetic predictor for treatment response. The GSK3B gene variation may be a key molecular switch related to the clinical effectiveness of lithium.
Researchers block single gene NF-êB to reverse aging in mouse skin, demonstrating that aging is a result of an active genetic program. The study sets the stage for future genetic age-intervention therapies.
Dr. Bridges' group successfully demonstrates marker genes can be efficiently inserted into heart muscle cells in large animals using a novel surgical procedure and specially designed hardware. The approach has the potential to develop new treatments for various heart muscle diseases causing heart failure, with preliminary success showi...
A recent study using PET scans shows that an experimental gene therapy can normalize brain function in Parkinson's patients. The treatment was found to alter brain activity in a favorable way, primarily affecting motor networks.
Researchers are conducting a phase 1 clinical trial using gene therapy to treat LCA2, aiming to restore vision in individuals with inherited blindness. The treatment involves injecting trillions of replacement genes into the retina, using an adeno-associated virus to deliver the therapeutic gene.
Researchers found that gene signatures can predict which chemotherapy regimens are most effective for breast cancer patients. The study showed a significant improvement in response rates when using gene signatures to select treatments, suggesting a potential breakthrough in personalized medicine.
Researchers found that antibody responses to viruses like measles and mumps remain protective for several decades, even for life. This means people may not need booster shots as frequently as previously thought, potentially reducing unnecessary vaccinations.
A Buck Institute study found that lithium increases lifespan in nematode worms by reducing the activity of a gene that modulates chromosome structure. The research highlights the potential for using C. elegans as a model organism to study drug toxicity and genetic impacts of compounds.
Gene therapy administered intravenously protected healthy tissues in mice during whole-body irradiation, with 90% survival rate at 30 days. The therapy offered no harm to systemic function and could potentially provide wide-reaching public protection in radiation emergencies.