Researchers successfully treated genetically modified mice with Parkinson's disease using gene therapy, restoring motivation to eat and drink. The study suggests a potential treatment strategy for patients with Parkinson's disease, eliminating the need for daily L-dopa treatments.
Research reveals that the AHC gene, previously thought to program ovarian development, is actually crucial for male fertility. Males lacking this gene have defective testicles and are sterile. The study also found that overexpression of the AHC gene can interfere with the function of a key protein involved in testicular development.
University of Florida researchers have successfully developed a gene therapy approach that replaces a crucial protein protecting the lungs from an often-fatal lung-liver disease. The method uses an adeno-associated virus to incorporate the missing protein, achieving high levels in the bloodstream for therapeutic effect.
Scientists have developed a novel gene therapy system that uses a molecular rheostat to control the activity of therapeutic genes. This system allows for precise dosage control and can be achieved through oral administration of a simple pill.
University of North Carolina researchers have successfully used an antibiotic-like compound to externally regulate a gene implanted in the brain using AAV technology. This breakthrough suggests that gene therapy may eventually be feasible for human brain disorders such as Parkinson's disease and epilepsy.
Gene therapy aims to treat unborn children with genetic disorders like SCID and alpha-thalassaemia. The treatment involves introducing healthy copies of the defective gene into the fetus, but raises concerns about potential risks and long-term consequences.
Scientists at Thomas Jefferson University have demonstrated that a normally protective anticancer gene can block tumors from growing. Led by Dr. Antonio Giordano, they found that the damaged version of the gene, pRb2/p130, suppresses tumor growth in animal models, paving the way for gene therapy to treat lung cancer and other cancers.
Scientists have successfully administered gene therapy orally, effectively curing lactose intolerance in rats. The study demonstrates the potential for an oral delivery system to treat other diseases, such as diabetes.
A gene-transfer system invented by Dusty Miller has been recognized as the 'Nobel Prize of Applied Research'. The Retro-X System is a retroviral gene-transfer kit used in gene-therapy research, allowing for stable gene expression and long-term therapeutic results.
Researchers have successfully used gene therapy to slow down neurodegeneration in an animal model, offering hope for treatments of autosomal dominant disorders. The approach involves using ribozymes to destroy faulty messenger RNA, potentially treating conditions like Huntington's disease and ALS.
A University of Florida neuroscientist has discovered a mutant gene in chickens that helps researchers better understand a rare human eye disease called Leber's congenital amaurosis type I. The chicken model is being used to develop a form of gene therapy for treating the disease, which affects 100,000 to 200,000 people worldwide.
Researchers at Duke University Medical Center have shown that a new type of gene therapy can correct the defect in human blood cells, potentially providing a treatment for sickle cell anemia. The therapy uses ribozymes to edit defective RNA and could lead to a significant reduction in disease symptoms.
Scientists successfully used gene therapy to correct deafness in shaker-2 mice by identifying a recessive mutated gene responsible for the condition. The study's findings also revealed that the mutation affects a myosin enzyme involved in inner ear development, which is crucial for hearing.
Researchers demonstrate intratumoral delivery of the E1A gene, downregulating HER-2/neu expression and tumor responses. The study shows promise for using E1A gene therapy to treat a broad range of cancers.
Researchers found that localized gene therapy can produce widespread, systemic effects on rheumatoid arthritis. The study used an adenovirus to carry therapeutic genes for altered cell receptors, which lessened inflammation and cartilage destruction in both diseased and distant joints.
University of Washington researchers successfully use a modified virus to perform novel gene replacement, targeting mutated genes with high accuracy. The new method achieves efficient gene correction, allowing for precise control over the expression of the corrected gene.
The American Society of Gene Therapy will hold its inaugural annual meeting from May 28 through May 31 in Seattle, featuring sessions on gene therapy vectors and various diseases. Over 1,500 scientists are expected to attend, with a focus on education and collaboration.
Scientists at Ohio University and Progenitor Inc. use a nonviral gene expression system to eliminate human cancer cells in animals, achieving a 60 percent tumor regression rate. The T7 system allows for transient gene expression and has been shown to be effective against various types of cancers.
Researchers at Ohio University develop a faster gene function identification system using a nonviral gene expression system in zebrafish, reducing identification time from three years to two days. The technique allows scientists to study gene function without destroying the gene, making it valuable for studying genetic disease.
Researchers at Thomas Jefferson University are conducting a gene therapy trial for glioblastoma, a form of brain cancer. The trial aims to delay disease progression and improve quality of life for patients, offering a potential cure for the deadliest type of brain tumor.
A new delivery system may allow for more precise control over where new genes are inserted into an organism's chromosomes, improving gene therapy. The approach uses parvoviruses to target specific locations on the chromosomes, reducing the risk of genes causing harm or functioning poorly.
A Phase 1 study of BRCA1 gene therapy in patients with ovarian cancer demonstrated minimal side effects and a significant tumor reduction in three participants. The researchers found that the vector was not immediately destroyed by the body's immune system, allowing it to transfer the BRCA1 gene into cancer cells.
Researchers successfully used gene therapy to grow new blood vessels in eight people with obstructed leg circulation due to atherosclerosis. The therapy, called therapeutic angiogenesis, helped improve blood flow, reduce pain and gangrene risk, and even prevented amputations.
Researchers successfully transferred a normal human beta globin gene into mice bone marrow cells, achieving long-term expression and high levels of production. This breakthrough holds promise for the treatment of sickle cell disease and beta thalassemia.
University of Michigan scientists develop viral vector delivering dystrophin gene to adult mice with muscular dystrophy, inducing high levels of normal dystrophin protein for several months. The breakthrough could pave the way for effective gene therapy treatment for Duchenne muscular dystrophy.
Scientists at UT Southwestern Medical Center have developed a system in mice where the level of a genetically engineered protein responds to inflammatory signals. This method of gene therapy has great potential for treating chronic relapsing and remitting inflammatory diseases, and may allow for targeted delivery of anti-inflammatory t...
Researchers developed an iNOS gene therapy that prevents chronic organ rejection and transplant arteriosclerosis in animal models. The study found that the gene therapy suppressed the disease process by triggering production of nitric oxide, which blocks blood flow in transplanted organs.
Gale and doctoral student Steve Kogan used discourse analysis to examine a therapy session conducted by Michael White, a renowned therapist influenced by postmodern thinking. The study found that the therapist successfully managed the talk to make each participant central and accountable, promoting more transparent interactions.
Researchers have developed a gene therapy technique that restricts the activity of genes used for gene therapy to specific cell types, bypassing safety hurdles. This discovery could lead to targeted treatment of cardiovascular diseases and other disorders.
A new gene, AIB1, has been discovered in breast cancer cells that amplifies their growth. The gene is part of a family known as SRC-1 and interacts with steroid hormone receptors, enhancing tumor cell growth.
Researchers found that mice with the human gene Bcl-2, which protects against cell death, developed ALS significantly later and lived longer than those without. The study suggests that gene therapy could delay ALS onset and lead to the development of drugs mimicking protective genes.
Researchers discovered that abnormal expression of the WT1 gene is linked to nearly 70% of breast cancers, which may lead to new treatments. The study suggests restoring functional tumor suppressor genes like WT1 could help combat breast cancer.
Researchers have identified an inactive gene that contributes to cancer development when it fails to work in harmony with the tumor-suppressing p53 gene. The findings suggest that the bax gene can act as a tumor suppresser, and its dysfunction may lead to increased cancer cell death and tumor growth.
Researchers at University of Pittsburgh Medical Center have made significant progress in treating rheumatoid arthritis using gene therapy. They successfully expressed the interleukin-1 receptor antagonist (IL-1Ra) gene in synovial cells within joints treated with the approach.
Researchers at Ohio University have developed a nonviral gene therapy system that can express genes outside the nucleus of a cell, making it easier to deliver disease-fighting genes to tumor cells. This system has been successfully used in cell cultures and mouse models to promote anti-cancer activity.
Researchers at Stanford University and their colleagues have isolated a single gene, fru, that controls most aspects of male sexual behavior in adult fruit flies. The gene governs the fly's courtship ritual, including its courting song and ability to recognize females.
Researchers at Penn State have discovered a new gene called KiSS-1 that suppresses the metastasis of melanoma in laboratory mice. The gene, located on chromosome 1, reduces the spread of melanoma by at least 50 percent of the time and may be the most potent gene to block or suppress metastasis in human cancer.
Researchers successfully used a harmless virus to deliver a normal gene into muscle cells in animals and humans, producing the enzyme for at least three months. This breakthrough could lead to a single treatment for children with Pompe's disease, an inherited fatal heart condition.
A study found that certain genetic variants next to the human obesity (OB) gene are associated with obesity in young women, as well as depression and anxiety. The results suggest a direct link between the OB gene variants and depression, rather than just secondary effects.
A University at Buffalo study finds that certain genetic variants near the human obesity gene are associated with obesity in young women, as well as depression and anxiety. The study also suggests that these genes may contribute to the development of behavioral disorders in young women.