Articles tagged with Gene Therapy
Researchers identified 124 genes whose changes in expression differed depending on the treatment regimen, providing new clues on how medications work and how cancer cells protect themselves. The study's findings will help design more rational and effective ways to use drugs for deadly childhood leukemia.
Researchers have developed a new method using titanium oxide nanocrystals to deliver genes into cells, potentially overcoming current limitations of gene therapy. The nanocomposites can separate when exposed to light or x-rays, allowing for targeted gene delivery.
A France-based research team has discovered the gene responsible for Hutchinson-Gilford Progeria, a disease that causes young victims to age five to ten times faster than normal. The discovery is a critical step toward developing therapies for the disorder and programs to screen individuals for the defective gene.
Researchers at Michigan State University have identified a gene responsible for Smith-Magenis Syndrome (SMS), a rare genetic disorder. The discovery suggests that primarily one gene contributes to the phenotype, rather than multiple genes as previously thought.
Researchers found that gene transfer by adenovirus is less efficient in aging rat heart cells, highlighting the need for new treatment strategies. Boosting integrin expression may help overcome this hurdle, enabling low-dose gene therapy to deliver beneficial genes.
The Alliance has awarded grants to Dr. Andrew Davidoff, Dr. Thomas Griffith, and Dr. Jeffrey Bartlett to develop gene therapies targeting neuroblastoma, prostate cancer, and ovarian cancer. The research holds promise for improved quality of life and reduced side effects for cancer patients.
A decade-long analysis of 500 families with Alzheimer's Disease suggests a future of early prediction and prevention. The findings lead to a new vision of medical care, applicable not only to Alzheimer's but also to other genetic disorders.
A team of scientists from the University of Wisconsin-Madison has developed methods for recombining DNA segments in human embryonic stem cells using homologous recombination. This technique allows for manipulating any part of the human genome to study gene function and mimic human diseases.
The discovery of neurofibromin regulation may help develop targeted therapies to block neurofibromin degradation in NF1 patients and treat cancers with amplified Ras activity. Nf1 heterozygous cells show increased sensitivity to growth factors, suggesting even diminished neurofibromin levels can affect normal cell behavior.
Purdue University researchers have discovered how RNA molecules bind energy-bearing ATP molecule, enabling physical work and potential applications in nanotechnology. The discovery sheds light on RNA's role in creation of living things and may unlock new methods for delivering therapeutic molecules.
Limited availability of genetic data hampers scientific progress in finding new therapeutic targets. The Lancet editorial emphasizes the need to share accumulated evidence and re-evaluate reporting standards.
USC researchers have successfully used gene therapy to prompt mouse cells to produce human collagen, a crucial step towards treating patients with dystrophic epidermolysis bullosa. This breakthrough could lead to the development of new treatments for this debilitating skin condition, which causes blisters and permanent scars.
Researchers at Purdue University have simplified the outer shell of the Ebola virus, making it easier to produce in a lab and delivering genes to defective cells. This breakthrough could lead to treatments for diseases like cystic fibrosis and lung cancer.
Researchers at Vanderbilt University Medical Center have developed a novel gene therapy approach that repairs messenger RNA, which could lead to effective treatments for inherited diseases. The method uses ribozymes to correct defective genes and has shown promising results in animal models.
Researchers at Ohio State University successfully tested micro-sized gelatin particles that may deliver therapeutic genes to the glomerulus in the kidneys. The biodegradable particles dissolved quickly enough to pass through the glomerulus within 10 hours, suggesting they could be useful in transferring genes to tissues.
A new gene therapy approach has been shown to virtually eliminate cancerous tumors in mice by boosting their immune systems. The treatment, which blocks a key immunosuppressor called TGF-beta, resulted in 70-80% survival rates in mice with melanoma and prostate cancer.
A new gene profiling technique could help predict which breast cancer patients will respond to hormonal therapies and which will require additional chemotherapy. The study aims to identify the cellular programs that must be engaged or shut off for aromatase inhibitors to be successful.
New research from University of Minnesota presents promising findings on unrelated donor transplants, including a study showing improved survival rates and quality of life for leukemia/lymphoma patients. Another study explores the use of bone marrow-derived stem cells to treat Parkinson's disease.
Researchers have found that blocking myostatin in mice with muscular dystrophy improves muscle function, providing a potential new treatment for the condition. However, further studies are needed to address concerns about the approach's limitations and potential side effects.
Researchers have developed a novel system of gene therapy that targets lung stem cells to treat cystic fibrosis. By using a natural compound found in the lungs, the therapy enables airway cells to take up the therapeutic gene and shows substantial recovery from the disease defect.
As new genetic components are identified, clinical services must adapt to cope with the increasing demand. The role of genetic information in managing inherited cancers is becoming increasingly important, enabling tailored therapies for individual patients.
Researchers have developed a gene therapy technique that integrates DNA without using viral vectors, inserting the DNA at known locations. The technique successfully integrated a gene coding for Factor IX into mouse DNA, making 12 times more protein than control mice.
A new study using gene therapy has shown promise in treating Parkinson's disease by 'resetting' overactive brain cells. The therapy may arrest or delay disease progression, offering a safer and more powerful alternative to current treatments.
Researchers successfully used gene therapy to change the brain's circuitry in rats with Parkinson's disease, producing nearly 70% fewer symptoms. The treatment involves altering glutamate and GABA signaling pathways to protect dopamine neurons from deterioration.
A team led by Purdue University's David Sanders has developed a promising system to deliver genes to diseased liver and brain cells using a modified Ross River Virus (RRV) as a Trojan horse. This approach avoids the harmful side effects associated with other retroviruses, bringing gene therapy closer to treating human diseases.
Researchers at the University of Florida have demonstrated a novel approach to overcoming gene therapy hurdles by attaching viruses to tiny manufactured balls coated with iron oxide. The microspheres can be targeted using magnets, allowing for more efficient delivery of corrective genes to specific organs or tissues.
Researchers develop novel approach to treat thalassemias by blocking aberrant RNA splicing. The new virus effectively repairs existing RNA, restoring correct hemoglobin production and offering a lifeline for patients.
Researchers have successfully delivered the full-length dystrophin gene to mice with muscular dystrophy using stripped-down vectors, restoring normal muscle function. The breakthrough could pave the way for human clinical trials to assess the safety of this method in patients.
A novel gene therapy technique developed by Stanford researchers allows for the integration of large genes into human chromosomes, enabling continuous production of proteins. This technique has the potential to treat a variety of diseases, including Duchenne's muscular dystrophy and cystic fibrosis.
Researchers have developed a gene therapy approach to treat mucopolysaccharidosis VII, a disorder affecting multiple organ systems, in dogs. The treatment involves four intravenous injections of a retroviral vector expressing canine beta-glucuronidase, resulting in normal enzyme activity and near-normal mobility in treated dogs.
Researchers developed a gene therapy combining radiation therapy and cisplatin to target tumor cells, resulting in significant regression with no additional toxicity. The treatment uses a modified cold virus to insert the TNF gene into tumor cells, enhancing anti-cancer effects.
The study discovered a novel gene, ADAM33, linked to asthma susceptibility and airway changes in asthmatic patients. This gene is involved in generating bronchial hyperresponsiveness and may underlie abnormalities in asthmatic airway function.
A study identified three gene expression patterns correlated with survival after chemotherapy, which outperformed the International Prognostic Index. The patterns revealed aspects of tumor biology affecting treatment response, including immune system involvement and tumor cell division.
A new gene therapy protocol has successfully treated a child born without an immune system, reversing severe combined immunodeficiency. The procedure improved genetically altered stem cells, giving them a biological advantage and allowing them to prevail over the abnormal cells.
Researchers have successfully used gene therapy to treat two young children with ADA-SCID, a rare form of SCID that requires regular injections of the bovine form of ADA enzyme. The new method involves removing bone marrow cells and engineering them to produce healthy immune cells, offering a potentially lower-risk alternative to bone ...
Biologists found that nematodes use a sophisticated mechanism to render transposons harmless, preventing them from making proteins and jumping through DNA.
Researchers at Georgetown University Medical Center have developed a germ cell line derived from spermatogonia, offering a basic tool for studying male infertility and testicular cancer. This cell line exhibits characteristics of normal adult spermatogonial stem cells and differentiates into advanced germ cell types upon stimulation.
Researchers created a mouse model of hair loss syndrome, which sheds light on the complex interactions between genes and their effects on human diseases. The study found that genetic background plays a significant role in determining the severity of the condition, and could potentially inform gene therapy approaches.
A recent study, TRAFFIC, found that intra-arterial infusions of recombinant fibroblast growth factor-2 (rFGF-2) improved exercise capacity in patients with moderate-to-severe intermittent claudication. Peak walking time increased by 1.77 minutes with single-dose rFGF-2 and 1.54 minutes with double-dose rFGF-2.
A new gene therapy tool has shown promising results in treating mice with hemophilia A by correcting the faulty factor VIII gene. The technology uses RNA trans-splicing to produce normal factor VIII protein, offering a potential cure for this genetic disorder.
Dr. Katherine High, a leading gene therapy researcher, has been chosen by the Howard Hughes Medical Institute for her groundbreaking work on treating hemophilia B. Her research holds promise for delivering therapeutic genes directly into patients' cells to improve blood clotting and quality of life.
Researchers have discovered the location of the genes that can cause heart attack, a breakthrough that could lead to new means of prevention and treatment. The study used genetics maps to scan chromosomes and found regions on chromosomes two, three, and 20 linked with heart attacks.
Scientists have found a way to activate the utrophin gene, which can help compensate for the lack of dystrophin protein in muscles affected by Duchenne's muscular dystrophy. The study suggests that over-producing utrophin may be a viable alternative to adding a working copy of the dystrophin gene through gene therapy.
Research findings link insulin-like growth factor I (IGF-I) to decreased lung cancer risk when bound by IGFBP-3. Environmental factors influence DNA methylation patterns in tumor suppressor genes, which may impact cancer incidence. Electro-gene therapy enhances IL-12 protein delivery, triggering long-lasting antitumor responses.
Researchers have successfully used a virus that causes warts to restore LDL receptor function in cells with defective genes, offering new hope for treating familial hypercholesterolemia. The next step is testing this technique in animals and humans to determine its safety and effectiveness.
Researchers at UT Southwestern and M.D. Anderson have identified eight genes on chromosome 3 that act as tumor suppressors in lung cancer. These genes were found to inhibit tumor growth and induce cell death in human lung cancer cells, suggesting a potential new approach for treating the disease.
A new type of dendritic cell has been found to prevent diabetes in mice by causing T cells to die rapidly. In a study, researchers treated mice with this special dendritic cell and found that half remained diabetes-free at one year.
Researchers analyzed participants' CETP genes to find genetic markers for heart disease. They discovered a pattern of gene variations called haplotype, which showed a significant association with the person's response to statin therapy.
Researchers discovered two mechanisms by which adenoviruses establish persistent infections: interfering with apoptosis signaling and preventing apoptosis through the Receptor Internalization and Degradation complex. This knowledge could improve the usefulness of adenovirus vectors in gene therapy.
The Lancet Neurology aims to report the most important advances in neurology, predicting risk of neurological conditions through genetics and imaging. Experimental therapies like gene therapy and stem cells offer potential for treating neurological disorders.
A study by UC Berkeley's Bruce N. Ames found that megavitamin therapies can effectively treat over 50 genetic diseases, mostly rare metabolic disorders due to defective enzymes. High-dose vitamins, particularly B vitamins like niacin and thiamine, may also improve metabolism in older populations.
A team of researchers at the University of Pittsburgh has discovered a gene mutation responsible for hereditary gingival fibromatosis (HGF), a rare condition characterized by gum overgrowth. The discovery may lead to new therapies, including tissue engineering, and could have implications for treating common dental problems like period...
Researchers successfully treated rats with chemical-induced Parkinson's disease using gene therapy, restoring normal limb movement. The treatment involves delivering two corrective genes into a specific brain region to prime the production of dopamine, which coordinates limb movements.
Researchers found that women with a common mutation in the estrogen receptor alpha gene had dramatic increases in high-density lipoprotein (HDL), or the 'good' cholesterol. This discovery suggests that genetic testing may help doctors make better decisions about hormone replacement therapy for preventing heart disease.
Researchers have discovered at least 100 genes that show significant differences in activity between non-invasive ductal carcinoma in situ (DCIS) and invasive breast cancer (IBC). The study's findings suggest that defects in these genes may play a role in the progression from non-invasive to invasive cancer.
A new intracoronary gene therapy has shown promising results in treating stable angina, a condition affecting nearly 7 million people nationwide. The study, led by the University of Vermont, involved 79 patients and demonstrated improved exercise ability compared to placebo groups.
Researchers successfully combined therapeutic cloning, embryonic stem cell differentiation, and gene therapy to treat a genetic immune disorder in mice. The study demonstrates the potential for nuclear transplantation therapy to correct genetic mutations and restore function in human patients.
Researchers have identified key genes associated with cardiac hypertrophy, hypertension, and dilated cardiomyopathy. Genetic testing is also being developed to predict susceptibility to coronary artery disease, offering new avenues for preventive medicine.
Gene therapy has shown promising results in hemophilia trials, with patients experiencing significant improvements in quality of life. The treatment involves inserting a normal version of the defective gene to produce clotting factor, and has been tested in small animals before being moved to human trials.
Researchers at Northwestern University have developed a gene therapy that reduces inflammatory cytokines involved in joint inflammation. The treatment, which uses the IL-13 gene, has been shown to suppress joint inflammation and prevent the development of arthritis in rat models.