Articles tagged with Gene Therapy
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 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 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 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.
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.
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.
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 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.
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.
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...
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.
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.
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...
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.
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.
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 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.
Researchers at Cedars-Sinai Medical Center have developed a novel gene delivery system that can sustain therapeutic expression in the central nervous system for up to 1 year. This innovative approach uses an adenoviral vector called HC-Adv, which is invisible to the immune system and renders previously used vectors ineffective.
Researchers have identified two genes, ARTS1 and IL23R, that increase the risk of developing ankylosing spondylitis, a common disease causing back pain and progressive stiffness. A potential treatment for Crohn's disease may also be applied to this disease, providing a major step towards being able to treat ankylosing spondylitis.
Researchers at Cincinnati Children's Hospital Medical Center found that Hypoplastic Left Heart Syndrome (HLHS) has high heritability and is likely caused almost entirely by genetic effects. Families with a child with HLHS carry a significant recurrence risk of HLHS or related heart defects.
Researchers at Children's Hospital of Philadelphia successfully delivered beneficial gene to entire mouse brain with one injection, correcting diseased areas. The technique has potential for treating rare genetic neurological disorders like Tay-Sachs disease and Sly syndrome.
Researchers at the University of Missouri-Columbia have identified 400 genes that can be manipulated to make chemotherapy drug cisplatin more effective. These genes, involved in sphingolipid metabolism, are linked to whether a tumor cell survives or dies after treatment.
New nonviral gene delivery systems utilizing transposons provide a safer alternative to viruses, with potential benefits for treating fatal conditions such as cancer. The technology has shown promise in achieving stable expression of genes in animal cells and minimizing insertional mutagenesis risk.
Researchers identified 17 genes linked to high-risk multiple myeloma, which can predict poor prognosis and guide therapy. The activity of these genes could help categorize patients' risk early, enabling personalized treatment plans.
A recent study found that a specific genetic variant affects smoking cessation treatment outcomes, with 45% of individuals benefiting from bupropion treatment and maintaining abstinence longer compared to those without the variant.
Gene therapy holds great promise but faces safety concerns due to virus-based delivery methods. MIT researchers have created biodegradable polymers that can deliver genes safely and effectively, showing promise for ovarian cancer treatment and other applications.
Researchers at Rensselaer Polytechnic Institute are developing a personal device to measure daily light intake and activity, predicting optimal timing for light therapy to synchronize the circadian clock. The device aims to alleviate psychosocial stress and sleep-related ailments caused by inadequate or irregular light exposure.
Researchers develop bacteria to deliver anti-cancer treatments by targeting oxygen-starved areas of tumors. The treatment shows effective results in experimental animals, offering a promising alternative to traditional cancer therapies.
Researchers at UT Southwestern Medical Center have discovered a single gene that controls fat formation in various organisms, including humans. The 'adipose' gene is believed to be a high-level master switch that determines whether the body accumulates or burns fat.
Researchers developed a genetic dosing model to predict optimal warfarin dose for patients undergoing orthopedic surgery. The model combines genotype information with clinical factors to estimate therapeutic dosage.
A research team has identified a genetic deficiency that causes severe blindness, specifically Leber Congenital Amaurosis (LCA). The discovery of the LCA5 gene and its protein lebercilin provides new opportunities for gene therapy, which could lead to the treatment of this disease in humans.
Researchers at Virginia Commonwealth University identified two key proteins, KLF2 and EKLF, that regulate overlapping groups of genes during red blood cell development. The study may lead to future gene therapies for sickle cell anemia and beta-thalassemia.
Researchers identified over 50 genetic abnormalities in sporadic ALS, providing expanded opportunities for developing treatments. The study's findings also uncovered genes controlling nerve adhesion, offering a major new avenue for ALS research.
Researchers developed a new vaccine strategy that uses a protein-free M. Tuberculosis strain to activate immune cells, providing increased protection against TB. A study found that vaccination with this strain provided better protection than the existing BCG vaccine.
A study published in the Journal of Clinical Investigation reveals that variants of the TCF7L2 gene increase susceptibility to type 2 diabetes by affecting insulin secretion in pancreatic islet cells. The findings provide a mechanism for genetic predisposition and suggest potential therapeutic targets for new treatments.
Researchers have developed a biodegradable nanoparticle delivery system that uses magnetic forces to target specific cells in the body. The system has shown promise in reducing cell proliferation and delivering anti-growth genes to stents, which could help maintain blood flow.
Researchers at Duke University Medical Center discovered a novel gene associated with increased susceptibility to multiple sclerosis, which increases risk by 30%. The gene variant affects the function of interleukin-7 receptor alpha, guiding immune system cell development and growth.
Researchers created a tunable genetic switch to regulate gene expression, overcoming limitations of existing technologies. The switch enables precise control over gene function, offering potential applications in basic research, gene therapy, and cell and gene therapy.
Researchers at WashU Medicine have identified hundreds of DNA elements that control when and where genes linked to blindness are turned on. These new elements can be used as switches to activate blindness therapies, offering hope for a potential cure for inherited blindness.