Articles tagged with Gene Therapy
Researchers developed a genetic blueprint that predicts response to two chemotherapy drugs and/or radiation therapy in esophageal cancer patients. The study found six gene variants associated with improved survival rates, with certain combinations more effective than others.
Researchers have discovered gene portfolios that can define a patient's likelihood of developing cancers, responding to treatment, and fending off malignancies. These 'Death from Cancer Signature' portfolios provide opportunities for early identification and targeted therapies, potentially improving five-year survival rates.
A breakthrough in gene therapy has successfully corrected hemophilia in laboratory animals by producing a normal level of clotting factor activity and preventing bleeding incidents.
Researchers test a new therapy for advanced melanoma, delivering a DNA plasmid through electroporation to stimulate the immune system. The results show high success in curing mice and preventing tumor regrowth.
Researchers at UT Southwestern Medical Center have developed a new gene therapy technique that uses homologous recombination to replace mutated genes in human immune cells, restoring both gene function and protein production. This approach has shown promising results in treating severe combined immunodeficiency disease (SCID) and may a...
Researchers at Duke University Medical Center have successfully reversed glycogen buildup in heart and skeletal muscle of genetically altered mice using a muscle-targeted gene therapy. This approach shows promise as a potential treatment strategy for Pompe disease patients who fail to respond to enzyme replacement therapy.
Researchers created a platform technology that can be adapted for various tissue types and preclinical uses, enabling real-time monitoring of gene expression. This approach uses magnetic resonance imaging (MRI) to non-invasively image cells at high resolution, providing insights into disease mechanisms.
The American Chemical Society's national meeting showcased groundbreaking research on various topics, including an antioxidant mixture that may prevent skin cancer, genetically engineered bacteria for mass-producing antibiotics, and novel gene therapy vectors to combat chronic pain and brain cancer. Researchers also discovered a potent...
Researchers found that the MIF gene is crucial for the development of type 1 diabetes. Specially bred mice without the gene failed to develop diabetes compared to those with it. The finding provides proof of concept for blocking the formation of this protein as a promising approach to fighting diabetes.
A new lab technique has identified high levels of pathogens in a hospital therapy pool, with
Researchers from the Forsyth Institute and University of Texas Health Science Center at San Antonio describe successful experiments in bioengineering mineralized tissues, including periodontal tissues and replacement tooth phenotypes. This breakthrough is supported by the National Institute of Dental and Craniofacial Research.
Researchers have successfully treated an animal model of alpha-mannosidosis, a rare genetic disorder that causes severe mental retardation and skeletal abnormalities, with gene therapy. The treatment restored damaged neurons and improved brain function in cats with the disease.
A team of biomedical engineers and chemists at Boston University developed a computational tool that predicts the molecular targets of drug candidates. The tool uses a combination of computational and experimental methods to decipher regulatory networks and predict gene and pathway targets for various drug treatments.
Researchers have developed a gene therapy that completely cures Crigler-Najjar syndrome, a liver disease, in rats using a specially designed adenovirus. The treatment has no long-term effect and poses no risk of cancer, making it a promising approach for treating other diseases with similar genetic deficiencies.
Scientists isolated immune cells from HIV-infected persons and cultured them with chemical compounds, producing the same changes as gene therapy. The findings offer a new approach to strengthen the immune system's capacity to stave off HIV.
A new study from St. Jude Children's Research Hospital found that both inherited genetic traits and tumor mutations can affect the response to treatment of leukemia. The research identified specific genes, such as GSTM1 and TYMS, that predict which children are at higher risk of relapse after initial therapy.
The Recombinant DNA Advisory Committee has played a central role in developing responsible research practices and establishing criteria for the safe use of recombinant DNA technology. The committee's work has enhanced opportunities within modern genetics and influenced the conduct of science globally.
UCLA researchers develop gene therapy approach using modified HIV to target P-glycoproteins on cancer cells, minimizing side effects and maximizing therapeutic effectiveness. The method shows promise for controlling cancer and other diseases.
Scientists prevented brain damage by producing therapeutic levels of GDNF, a protein that helps nourish brain cells, in small monkeys. The gene therapy approach aims to halt Parkinson's disease at its onset and treat devastating side effects.
A study led by William Giannobile at the University of Michigan showed that gene therapy involving BMP-7 can produce nearly 50% more supporting bone around dental implants. This breakthrough has potential to reduce the need for bone grafts in reconstructive surgery.
A phase II clinical research study uses VEGF-2 gene therapy to stimulate growth of new blood vessels, relieving angina symptoms. The study aims to improve quality of life for patients with refractory chest pain.
Researchers discovered a critical 'gatekeeper' function that prevents auto-antibody accumulation in lupus, which can be restored to reverse the disease. Increasing Fc receptor activity by 40% restored health in animal models of lupus.
Researchers found that 50% of c-MYC-induced mammary cancers persisted in mouse models despite c-MYC gene blockage. Residual cancer cells quickly recovered malignant properties after treatment and eventually progressed to independent growth states.
Professor Xiaolian Gao's research develops a technology to mass-produce multiple genes on a single chip, reducing costs and time factors by 100 times. This breakthrough has the potential to create complete functioning organisms, produce energy, neutralize toxins and make drugs.
Researchers discovered a jumping gene called Hermes, which creates changes in DNA similar to those created by the process behind antigen recognition. This discovery provides evidence that genetic processes behind antigen diversity may have evolved from the activity of a jumping gene.
A new study has provided proof of principle for a combined stem cell-gene therapy approach as a potential treatment for cystic fibrosis. The researchers were able to correct the CFTR gene defect using adult stem cells and ex vivo gene therapy, which may offer advantages in screening and long-term function.
A new gene therapy approach has successfully corrected immunodeficiencies in 17 of 18 patients with severe combined immunodeficiency disease. The treatment, which involves introducing a 'good' gene into stem cells, has shown clear and sustained clinical benefits.
Researchers develop gene therapy to treat XP, a devastating disease causing cancerous lesions on exposed skin. The treatment restores DNA repair mechanism, preventing cancerous growth and sunburn-like symptoms in mice.
Researchers at Cedars-Sinai Medical Center found that a genetically engineered virus delivering the protein hsFlt3L slowed tumor growth and increased immune cell production in laboratory rats with glioblastoma multiforme. The study may lead to a new treatment approach for patients with GBM.
Researchers at Thomas Jefferson University used gene therapy to restore heart function in rats with failing hearts, reversing cardiac dysfunction. The study found that delivering the human S100A1 gene improved heart muscle efficiency and reversed cardiac dysfunction.
Researchers found that viral delivery of a parkin gene protected dopamine-producing neurons from degeneration, offering new hope for treating Parkinson's disease. The study demonstrates the feasibility of gene therapy in a genetic model of the disease.
ALS researchers found that continuous administration of VEGF protein into the cerebrospinal fluid increases life expectancy in ALS rats. The treatment has shown promising results with a patient-oriented approach, allowing controlled doses to be easily administered.
A new gene therapy has successfully corrected the genetic defect responsible for Fabry disease in mice, producing a sustained level of enzyme activity and reducing fat buildup in target organs. The treatment, administered shortly after birth, could potentially lead to a one-time treatment option for humans.
Researchers identified a complete deficiency in the CD3 epsilon chain of the T cell receptor causing SCID, leading to normal B cells but no T cells. Early diagnosis via bone marrow stem cell transplantation can result in a survival rate as high as 97% for patients with this mutation.
A new gene therapy technique has successfully treated irregular heart rhythms in pigs, using a 'painting' method to deliver the therapy. The technique, which uses a plastic, gel-like substance to contain the gene therapy, shows promise for treating atrial fibrillation, a common heart condition.
Researchers at the University of Pennsylvania School of Veterinary Medicine have identified key growth factors essential for sustaining spermatogonial stem cells in culture. By understanding these factors, scientists hope to develop new fertility therapies and create sperm in vitro.
Researchers at Pitt University successfully reversed peripheral neuropathy in diabetic mice using gene therapy based on HSV vectors, delivering neurotrophic factors to affected neurons. The approach restores lost nerve endings and prevents progression of neuropathy, with promising results for potential human treatment.
Children's Hospital Boston geneticist Dr. Kunkel has made significant contributions to understanding muscular dystrophy, from identifying genes to developing new therapies. His work aims to find patterns common to all muscular dystrophies and those distinct to specific forms of the disease.
A recent study found that demethylation agents not only restart cancer-suppressing genes but also knock methyl groups off other genes, turning hundreds more off. The research raises concerns about the impact of these agents on gene expression and their potential to exacerbate cancer development.
Researchers at St. Jude Children's Research Hospital developed a gene vector that allows hematopoietic stem cells to produce fetal hemoglobin, reversing beta-thalassemia in mice. The technique uses a new vector with added regulatory elements to improve the expression of the gamma-globin gene.
NIST has awarded up to $80.1 million in funding for 32 new advanced technology program (ATP) projects, spanning various key technology areas including energy, medical diagnostics, electronics, and computer interfaces. The projects aim to improve productivity, facilitate trade, and enhance quality of life.
Researchers have identified a new region on the bcl-2 gene that regulates its expression and stability. This discovery may lead to the development of new cancer therapies by targeting the CA-repeated Region (CAR) to reduce bcl-2 levels in cancer cells.
Researchers have developed a novel gene regulation strategy using ribozymes, which can be controlled with virtually any drug, offering a safer alternative to existing methods. The technique enables the easy turn on and off of genes, allowing for potential applications in therapeutic and research settings.
A study funded by the Christopher Reeve Paralysis Foundation has identified novel potential therapeutic targets for spinal cord repair using microarray technology. The study analyzed 108 genes and found patterns of gene expression that could help researchers identify new treatments.
A study has identified two distinct subclasses of liver cancer with differing survival rates, providing new molecular insight into the pathogenesis of hepatocellular carcinoma. The findings suggest that gene expression profiles can accurately predict patient survival and may lead to the development of more targeted therapies.
Researchers will study 120 patients with chronic and aggressive periodontitis to identify molecular basis of disease and develop targeted therapies. The goal is to reduce risk of periodontitis and improve treatment outcomes for the leading cause of adult tooth loss.
A recent study found that genetic variations in the HLA and TNF gene regions are associated with response to treatment in early rheumatoid arthritis patients. Patients with two copies of the shared epitope were four times more likely to achieve significant improvement in disease activity compared to those with one or no copies.
Scientists uncover vital role of Periaxin gene in causing CMT, a disabling disease affecting 23,000 people in the UK. The new research provides insight into how nerves work and may lead to development of gene therapies to correct faulty genes.
Researchers developed a novel gene-based therapy that substantially decreased tumor size and eliminated cancer cells in experimental mice. The treatment, which uses recombinant adenoviruses encoding interferon-alpha, showed promise in responding to even resistant bladder cancer cell lines.
A gene associated with lupus has been identified, suggesting a potential breakthrough in diagnosing and treating the disease. The discovery could lead to earlier intervention and more effective therapies.
Researchers found that different retroviral vectors from ASLV, HIV, and MLV integrate into human chromosomes at varying sites. The study suggests ASLV may be a promising candidate for human gene therapy due to its distinct integration preferences.
Researchers at Jefferson Lab are adapting detector technology to develop a new gene therapy technique for cystic fibrosis, which affects 30,000 Americans. The technology allows for successful imaging of gene transfer in mice, paving the way for clinical trials.
Researchers at UCSD and VA San Diego Healthcare System have developed a novel method of transcriptional gene silencing in the nucleus, utilizing short interfering RNA (siRNA). This approach may provide a long-lasting solution for gene regulation, with potential applications in treating diseases such as cancer and HIV.
Researchers have developed a novel gene therapy that combines a therapeutic gene with a genetic sensor to protect the heart from damage caused by reduced blood flow. The therapy, which was tested in rats, showed a significant reduction in tissue death and damage compared to untreated animals.
Researchers have identified nearly 100 genes linked to cancer, shedding light on the processes driving disease progression. The study highlights the potential of targeted therapies and early detection methods to manage cancer.
Researchers have successfully delivered a mini-dystrophin gene to all skeletal and cardiac muscles of an adult mouse with muscular dystrophy, reversing the disease. The breakthrough uses a safe and simple method to target muscle cells without triggering an immune response.
Researchers found genetic mutations in fibulin genes, specifically FBLN5, that could contribute to AMD. However, these changes were not statistically significant, and the study highlights the importance of precise search methods for genetic causes.
A LSU vet school professor has successfully treated a dog with cancerous tumors using gene therapy, which helps the body develop an anti-tumor memory. The treatment involves electroporation and cytokine gene therapy, resulting in the elimination of smaller tumors and reducing the size of larger ones.
OHSU researchers are studying vaccination methods for seniors and children, with a focus on developing protection against infectious diseases. The study aims to understand immune system changes that occur with aging and develop methods to manipulate the system for better protection.
Using guinea pigs, researchers increased production of G-protein Gem, which decreased calcium current densities by 30-90% and helped steady irregular heartbeats. Gene therapy shows promise as an alternative to calcium channel blockers with fewer side effects.