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 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.
A study by Dr Tom Barber and colleagues found a genetic link between the FTO gene and PCOS susceptibility. The researchers analyzed 463 PCOS patients and 1336 female population controls, discovering that variants of the FTO gene influence fat mass and PCOS-susceptibility.
A new synthetic gene circuit allows for precise dosing of gene expression in yeast cells, enabling accurate analysis of a gene's role in normal and abnormal cellular function. The circuit utilizes negative feedback loops to achieve a linear dose-response relationship.
Researchers have rediscovered a dormant human gene, IRGM, which was thought to be extinct for millions of years. The study, published in PLOS Genetics, reveals that the gene was resurrected through a complex series of structural events, suggesting its potential importance in fighting new or resistant infectious agents.
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.
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.
Patients with relapsed follicular lymphoma who continue on maintenance rituximab therapy after chemotherapy have better overall survival than those without treatment. Rituximab maintenance therapy was associated with a 40% improvement in overall survival relative to observation or retreatment.
A study published in JAMA found that using N-terminal brain natriuretic peptide (BNP) to guide therapy did not improve overall clinical or quality of life outcomes for older patients with heart failure. In contrast, the study showed improved survival rates and reduced hospitalizations for patients receiving BNP-guided therapy.
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.
Postmenopausal women with a deficiency in the CYP2D6 gene have a nearly fourfold higher risk of early breast cancer recurrence when taking tamoxifen. Mayo Clinic researchers recommend testing for the gene before starting therapy.
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.
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.
A study found that patients with locally advanced lung cancer who received proton therapy and chemotherapy had fewer instances of bone marrow toxicity compared to those receiving IMRT and chemotherapy. This suggests that proton therapy may be a more effective option for treating this serious side effect, enabling doctors to give higher...
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.
Research studies and clinical registry data suggest that triple antiplatelet therapy achieves greater platelet inhibition than conventional dual antiplatelet therapy in patients undergoing percutaneous coronary intervention (PCI) with drug-eluting stents. This leads to reduced early mortality and major adverse cardiac events without in...
Researchers have discovered a novel mechanism in gene expression where non-coding RNAs create access to DNA, allowing transcriptional activation proteins to initiate gene expression. This process involves the transient synthesis of non-coding RNAs that unfurl tightly wound DNA, enabling gene expression.
Researchers have identified a gene in rice that controls the size and weight of grains. The GIF1 gene is responsible for controlling invertase activity, which is essential for starch production. By overexpressing the GIF1 gene in transgenic lines of rice, scientists were able to increase grain size and weight.
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.
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.
Women with migraines, especially those with aura, may be at higher risk for heart disease and stroke due to a specific gene variant. A study found that women with this gene variant had more than three times the risk of cardiovascular disease, including four times the risk for stroke compared to those without the gene.
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.
Researchers have developed a novel gene delivery approach that targets smooth muscle cells in blood vessels, offering a new paradigm for cell-specific gene delivery. The approach uses an electric field to transiently permeabilize plasma membranes and a specific DNA sequence to control nuclear entry.
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.
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.
A new analysis suggests that healthy postmenopausal women with normal blood cholesterol levels are at no increased short-term risk for heart attack when taking hormone therapy. Women with a low LDL to HDL ratio have an even lower risk of heart attack or death from hormone therapy.
A new study found that patients receiving budesonide/formoterol combination therapy reported significantly greater satisfaction with symptom relief, dosing management, and timeliness of medication effects. In contrast, patients treated with fluticasone/salmeterol combination therapy experienced lower levels of satisfaction in these areas.
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.
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.
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.
Researchers found that hormone therapy started around menopause onset may have similar benefits as those without risk factors for heart disease. The study suggests that current guidelines may need to be revised for younger women taking hormone therapy.
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.
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.
Researchers have identified a plant gene cluster that produces a triterpene compound, previously unknown in plants. The study reveals how plants can assemble beneficial gene clusters to produce valuable natural products.
Researchers investigated how inheriting different combinations of gene variants impact depression risk in normal subjects. They found that the SLC6A4 serotonin transporter gene interacts with the BDNF gene to regulate emotion regulation system development, revealing individual genes are not isolated risks.
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 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 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.
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.
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 found that a short course of hormonal therapy before and with radiation therapy slowed cancer growth by up to eight years, particularly the development of bone metastases. This delayed cancer progression increased survival rates among older men with potentially aggressive prostate cancer.
Researchers have successfully transferred a new gene to cancer patients via their own stem cells, aiming for stronger treatment with less severe side effects. The study found that six out of eight patients had stem cells carrying the MGMT gene in their blood or bone marrow after treatment.
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.
A 24-week course of interferon-alpha therapy after curative treatment for HCV-associated HCC significantly reduces tumor recurrence rates and improves patient survival compared to those without interferon therapy. Patients who achieved a sustained virological response also exhibit improved liver function and prolonged overall survival.
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.
A study conducted at Wake Forest University Baptist Medical Center found that early mobility therapy in ICU patients reduced length of stay by an average of three days, compared to those who did not receive the therapy. The therapy was provided by a mobility team and proved safe, with no additional costs incurred.
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.
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 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.
Researchers at the University of Texas M. D. Anderson Cancer Center have developed a gene therapy that selectively targets and eliminates pancreatic cancer cells, inhibiting metastasis and prolonging survival with minimal toxicity. The therapy, called VISA-BikDD, has shown significant promise in preclinical trials, with treated mice su...