Research on treating disease by editing or delivering genes. Covers sickle-cell gene therapy, gene-editing nanoparticles, mRNA delivery, and blood-cell-inspired carriers.
Researchers identified genetic signatures that predicted patient prognosis and response to treatment, outperforming existing clinical markers. The technique of DNA microarray analysis holds promise for developing new prediction methods, sparing patients from unnecessary treatments.
Researchers develop gene therapy that increases atrial natriuretic peptide production to control blood pressure without severely reducing it. The therapy successfully maintained healthy blood pressure levels for 125 days in mice.
A study found that certain gene variants, specifically ADRB2 genotypes, are associated with a higher risk of mortality in patients taking beta-blocker therapy for heart conditions. The researchers identified specific genotype groups at high risk for long-term mortality and suggested pharmacogenetic targeting to improve ACS care.
A recent study found that variations in the beta-2 gene significantly determine length of survival for acute coronary syndrome patients on beta-blockers. High-risk groups had a five times higher risk of death, while low-risk groups had a six percent mortality rate after three years.
The Phase I trial demonstrated the treatment's safety and efficacy in advanced Parkinson's disease, with statistically significant improvements in motor function and activities of daily living. The gene therapy approach offers a simpler alternative to deep brain stimulation, avoiding devices and requiring local anesthesia.
The Mayo team invented a simpler method for using modified viruses to transport therapeutic genes to tumors. They demonstrated that retrovirus particles could successfully attach to T cells and safely hitchhike to reach tumors, transferring genes that infected cancer cells.
Researchers have developed RNA nanoparticles that can carry multiple therapeutic agents into specific cancer cells, where they can halt viral growth or cancer progression. The tiny particles are assembled from three short pieces of ribonucleic acid and possess the right size and structure to gain entry into cells.
Researchers found that smokers with specific genetic variants were more likely to abstain from smoking if using Zyban, while those with other variants had higher quit rates with nicotine replacement therapy. The study suggests that genetic information can be used to select the most effective treatment for individual smokers.
Researchers at the University of Iowa developed a new gene therapy vector that can effectively deliver therapeutic genes to liver cells, converting Hemophilia A from a severe to a mild form in mice. The correction lasted 30 weeks and showed promise for reducing bleeding episodes in people with hemophilia.
Researchers at the University of Pittsburgh Medical Center have successfully treated animals with muscular dystrophy using a miniature gene and new systemic approach, demonstrating significant improvements in muscle function and longevity. The treatment involves delivering a mini-agrin gene via adeno-associated virus vectors, restoring...
Researchers developed a large brain tumor model and tested a genetically engineered virus delivering two proteins to the brain. The findings show that combined RAdTK and RAdFlt3L gene therapy eliminated glioblastoma multiforme in lab rats, increasing survival rates significantly.
A collaborative project developed a way to study the function of genes in mice and humans using a moveable genetic element from moths. The technique, called piggyBac, allows for efficient genetic manipulation in vertebrates and mammals, enabling researchers to systematically understand the functions of mammalian genes.
Researchers at UCLA have identified Rpe65 as a crucial enzyme in the regeneration of rhodopsin visual pigment in the retina after light exposure. This breakthrough could lead to a gene therapy cure for Leber congenital amaurosis, an inherited disease causing up to 20% of childhood blindness.
Researchers have achieved a cure for haemophilia in dogs using gene transfer strategies. The study identified key problems to overcome before applying this approach in humans, including mode of delivery.
Scientists at the University of Florida have developed a gene therapy that prevents blindness in mice with retinoschisis, a rare genetic disorder. The treatment involves injecting a healthy version of the human RS1 gene to prevent cyst formation and restore vision in affected eyes.
Scientists have discovered a way to treat Huntington's disease by targeting multiple proteins and genetic pathways simultaneously. This breakthrough has the potential to provide new hope for patients with this devastating neurodegenerative disorder.
A recent study suggests that Cyr61 gene therapy may be effective in treating severe myocardial ischemic disease by inducing the growth of new blood vessels. The research found potent stimulation of myocardial angiogenesis, a novel gene therapeutic approach, using Cyr61 in a porcine model of chronic myocardial ischemia.
Researchers have successfully applied a novel PET/CT technique to characterize the effects of a gene therapy intervention in animal models, demonstrating its potential for improving cardiac health. The study showed that successful transfer of a VEGF gene resulted in increased tissue perfusion without impairing cardiac function.
Researchers successfully introduced a gene that blocks joint inflammation, reducing IL-1 and IL-6 production. The study's nine patients showed no clinical side effects after five years, paving the way for new gene-based therapies.
The researchers developed a nebulized and inhaled gene therapy that reverses PAH in rats by inhibiting Survivin, a protein driving excessive cell growth. The therapy improves heart function and survival rates, offering promising avenues for human PAH treatment.
Researchers used disabled retroviruses to discover genes that increase the multiplication rate of stem cells, which could improve regenerative medicine. The study found that these genes can be used to enhance the fitness of stem cells, potentially leading to new treatments for inherited blood disorders.
Researchers use ferritin as a reporter gene to track genetic modifications in tumor cells, enabling monitoring of gene therapy's progress. This technique has far-reaching implications for diseases like diabetes, where genes can be tagged and tracked using MRI.
Researchers discovered a genetic link between a mutation and vitamin B6 responsiveness, enabling a genetic test for guiding treatment. The disease causes kidney stones or failure if left untreated, but early diagnosis with this test can maximize successful treatment outcomes.
Researchers identified 18 genes that predict which patients will respond to Hepatitis C treatment, offering a potential simple blood test for early diagnosis. The study found that these genes are key to the body's ability to eliminate the virus.
Researchers used pulsed-HIFU to treat tumors in mice with a reporter gene, achieving nine times higher uptake compared to untreated tumors. The technique has potential for enhanced delivery of therapeutic genes across various tissues without scars or needles.
A study by University of Florida researchers found that genetic variations can determine how well heart failure patients tolerate beta-blockers. The findings suggest that personalized therapy based on genetic makeup could lead to safer and more effective treatments.
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.
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.
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...
A new lab technique has identified high levels of pathogens in a hospital therapy pool, with
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.
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.
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.
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.
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.
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.
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.
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.
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 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.