Articles tagged with Gene Therapy
The Phase I trial demonstrated statistically significant clinical efficacy and neuro-imaging results, with patients showing a 25% improvement in Unified Parkinson's Disease Rating Scale scores. PET scan data revealed a significant improvement in brain metabolism on the treated side of the brain compared to the untreated side.
Scientists have mapped a common form of Fuchs corneal dystrophy to chromosome 18, shedding light on its genetic origins. The discovery has implications for developing gene therapies to treat the condition.
Researchers have developed a gene therapy treatment that restored retinal function to near-normal levels and prevented degeneration in cones of mice with Leber congenital amaurosis. The study shows promise for potential human applications, offering new hope for individuals affected by this condition.
A recent study by Dr. Robert Koenekoop and colleagues has identified the CEP290 gene as the most common cause of Leber Congenital Amaurosis (LCA), a form of congenital blindness. The discovery could lead to improved screening and treatment options for affected children.
A Dutch researcher found that genetic characteristics, such as sweat production and brain receptor genes, can predict which drug works best for each patient. This information may lead to more effective treatment allocation for alcoholics.
Children's Hospital of Pittsburgh researchers aim to develop more effective treatments for patients with chronic lung diseases, including cystic fibrosis and idiopathic pulmonary fibrosis. The $12.8 million NIH grant will support the development of new diagnostic methods and therapies.
A new gene expression-based chemical genomic approach has identified potent inhibitors of androgen receptor signaling, including celastrol and gedunin, which may overcome prostate cancer treatment resistance. Additionally, rapamycin has been found to potentially reverse glucocorticoid resistance in childhood acute lymphoblastic leukemia.
Researchers developed a gene transfer method using the mutant form of apolipoprotein A-I (Apo A-I Milano) to treat vascular inflammation and plaque buildup. The study found that Apo A-I Milano gene transfer resulted in a 65% reduction in plaque buildup, compared to a 25% decrease with the normal HDL gene.
Researchers found that piggyBac transposon is five to 10 times better than other circular pieces of DNA at making a home and difference in several mammalian cell lines. This could lead to safer and more efficient gene delivery for therapeutic applications.
Researchers found that increasing leptin production in the hypothalamus helps regulate insulin secretion, keeping blood glucose levels normal. Gene therapy successfully reversed type 2 diabetes in diabetic mice, even after a high-fat diet.
Researchers found that patients with advanced squamous cell carcinoma of the head and neck whose tumor samples over-expressed p53 protein were significantly more likely to respond to Advexin therapy than those with low p53 protein. The study suggests that p53 overexpression may be a predictive biomarker for Advexin efficacy.
A study by Purdue University researchers has identified unusual genetic traits in voles that challenge current scientific understanding. The vole's unique genetic makeup, including its ability to insert DNA into the nucleus, could have important implications for human genetics and gene therapy.
Researchers found that a single genetic assay can predict response to different targeted therapies in gastrointestinal stromal tumors. Patients with exon 9 mutations or wild-type genes may benefit more from Sutent, while those with exon 11 mutations may require longer Gleevec treatment.
Researchers discovered that viruses can infect cells more efficiently by attaching to different carbohydrates on the cell surface. This finding helps explain how flu and other viruses evade the immune system and may be useful for developing gene therapies for cancer and brain diseases.
Researchers at Virginia Tech have designed polymer macromolecules as effective gene transfer agents, overcoming the need for foreign DNA and viruses. The study's findings focus on the structure of these molecules, which can control their ability to transfer genes across cell membranes.
A Scripps Research Institute team developed compounds that reactivated the frataxin gene in blood cells from 13 Friedreich's ataxia patients, with one compound producing full reactivation in 100% of cells tested. The findings offer a potential therapeutic avenue for the disease, which affects 1 in 20,000 people in the US.
This study confirms the existence of immunological synapses, microanatomical structures similar to those of nerve cells, in laboratory rats. The findings settle controversy over their existence and functional significance during antiviral immune responses, paving the way for further research.
A $14.6 million NIH grant will fund an international, multidisciplinary effort to leverage recent genetic discoveries into possible treatments for AMD. The investigation aims to explore the role of complement factor H and factor B genes in the immune system and develop diagnostic tools and treatments.
Researchers at St. Jude Children's Research Hospital developed a mouse model that explains why gene therapy treatment caused leukemia in some severe immune deficiency patients with XSCID. The study found that the disease itself makes mice susceptible to cancer caused by gene therapy, offering hope for safe treatment.
A recent study has found that thrombotic complications occur in 5.2% of children with leukaemia, with the highest risk during antileukaemic therapy induction phase. The researchers have identified several factors associated with an increased risk of thrombosis and explored the role of genetic determinants.
Queen's University researchers found that two genes cooperate to protect aortic smooth muscle cells from death, pointing to new therapies for preventing heart attacks and strokes. The study's findings suggest that delivering the HO-1 gene using gene therapy may help stabilize plaques and prevent clogging in arteries.
Researchers used gene therapy to deliver glial-derived neurotrophic factor (GDNF) directly to the brain cells of mice with Huntington's disease, protecting neurons from degeneration. The study showed improved behavioral function and reduced symptoms in mice treated with GDNF, suggesting a new approach to forestall disease progression.
Researchers used gene therapy to treat ovarian cancer in a mouse model, achieving complete inhibition of tumor growth in some mice and significant inhibition in others. The treatment offers a promising alternative to harsh current treatments for this deadly disease.
Researchers developed gene therapy using manganese superoxide dismutase to protect mice from radiation damage. The treatment showed no changes in body weight, little bone-marrow damage, and longer lifespan in mice exposed to varying doses of whole-body radiation.
Researchers used adeno-associated virus to insert genes for interleukin-4 (IL-4) or interleukin-10 (IL-10) into insulin-producing beta cells, preventing hyperglycemia in non-obese diabetic mice. Gene therapy shows promise as a viable method for preventing type 1 diabetes in genetically at-risk individuals.
A novel therapeutic approach has been discovered by the University of Manchester team, using a DNA-binding protein to stimulate genes and promote nerve growth. The preclinical results have been encouraging, with initial-stage clinical trials in the US showing promising progress in managing diabetic neuropathy.
Researchers at Stanford University School of Medicine have made significant progress in developing RNAi gene therapy, a promising approach to treating organ-wide diseases. However, they also encountered unexpected side effects, including liver toxicity in mice, which hindered their progress. Despite this setback, the team was able to o...
Researchers at the University of Florida have successfully restored vision to chickens with a genetic defect causing blindeness. Five out of seven treated chickens displayed near-normal visual behavior and responded to light, offering hope for an eventual vision-restoring therapy for children with inherited childhood blindness.
Researchers have successfully treated a chicken model of LCA1, a form of childhood blindness, using gene therapy. Six out of seven treated chicks developed sight, despite only a small percentage of receptor cells being infected by the virus. This study demonstrates the potential for gene therapy to treat this form of blindness in humans.
In-vivo gene therapy has successfully restored immune function in three out of four dogs treated for XSCID. The treatment approach eliminates the need for ex-vivo cell manipulation and culture, potentially reducing adverse side effects and improving long-term efficacy.
Researchers have developed a drug that increases SMN protein levels in SMA patients, improving their quality of life. Another study shows promise for treating X-linked adrenoleukodystrophy (X-ALD) and Duchenne muscular dystrophy (DMD) with valproate and anti-sense molecules.
A new study by Harvard Medical School researchers found that visual stimulus turns up the expression of some genes and turns down others, shaping the brain. The study identified distinct sets of genes that respond to visual input at different ages, suggesting a more holistic view of gene function in neural development.
A recent mouse study led by the Salk Institute has discovered that healthy copies of the IL2RG gene used in X-linked severe combined immune deficiency (X-SCID) gene therapy can promote cancer development. One-third of mice treated with the gene developed lymphoma later in life.
Researchers have made progress toward a new form of light-activated gene therapy for cartilage repair, which is safe and compatible with existing surgical techniques. The therapy uses UV light to target specific cells and promote tissue growth, offering hope for treating knee injuries and potentially other conditions.
The landmark book celebrates the centennial of Alzheimer's discovery, narrating its evolution from a major cause of late-life dementia. The publication brings to life classic studies defining the disease research, featuring contributions from prominent researchers.
The study reveals high molecular heterogeneity in multiple myeloma, with new recurrent amplifications and deletions pointing to yet-to-be-discovered oncogenes and tumor suppressor genes. These findings define new disease subgroups with distinct clinical outcomes and potential therapeutic implications.
The study showed that SB-509 was safe and well-tolerated, with no dose-limiting toxicities observed. Improvements in pain, numbness, and neurological symptoms were seen in approximately 50% of patients, with significant anecdotal improvements reported in neurologic exam scores and electrophysiologic testing.
Researchers at Cedars-Sinai Medical Center have developed a novel gene therapy strategy that modifies the brain microenvironment to induce an effective immune response against deadly brain tumors and infections. The treatment uses Flt3 Ligand to recruit antigen-presenting cells, which can destroy tumors and combat brain infections.
Researchers discovered that human cells deficient in LRP6 become resistant to anthrax toxin, while antibodies targeting LRP6 protect cells from toxicity. This finding suggests potential new avenues for treating late-stage anthrax disease.
Scientists at the University of California, Santa Barbara, have created a new lipid molecule that delivers therapeutic genes directly to cells, potentially helping inherited diseases and cancers. The novel molecule has a tree-shaped headgroup and displays superior DNA-delivery properties.
A research group identified 679 genes differently expressed in responders to chemotherapy compared to non-responders. RT-PCR analysis validated the findings, confirming the differential expression of 22 genes. The study suggests integrating molecular networks could improve diagnosis and treatment of colorectal cancer.
Researchers replaced a faulty gene responsible for hemophilia with a transposon from fish, preventing profuse bleeding in neonatal mice. The approach holds promise for treating the most common form of hemophilia, affecting nearly 18,000 Americans.
Defibrillator therapy found to be beneficial in reducing the risk of sudden cardiac death and prolonging life among patients with high-risk genetic cardiac disorders. The treatment is associated with significant cost savings, ranging from $15,000 to $20,000 per quality-adjusted-life-year saved.
Researchers at Florida State University have identified a gene called BDNF as playing a key role in social aversion, leading to the development of a potential new treatment for depression. Long-term use of antidepressants was successful in reversing social withdrawal, but a gene therapy approach showed promise with fewer side effects.
Researchers have identified a key gene associated with rare but important pregnancy disorders, including molar pregnancies, spontaneous abortions, and stillbirths. The discovery has significant implications for developing new treatments and therapies for these conditions.
A new lentiviral vector combines multiple gene manipulation techniques to efficiently regulate gene expression in cells. This versatile tool has potential applications in studying human genetic diseases, cancer research, and tissue engineering.
A study of 626 patients found that Black patients on HAART had a lower risk of atheroma than White and Hispanic patients. Genetic variants were associated with dyslipidemia in Hispanic patients, but not in others.
Researchers at Northwestern University have developed a gene therapy that turns off the alpha-synuclein protein, a key player in Parkinson's disease. The therapy uses RNA interference to selectively disable the gene, leaving other genes unaffected, and has shown promising results in rat models.
A saltwater aerosol solution has been found to restore the thin lubricant layer on airway surfaces, promoting mucus clearance and reducing lung damage in cystic fibrosis patients. The therapy is inexpensive and shows promise for slowing disease progression and improving patient outcomes.
The study illustrates 12 disease pairs and their therapeutic implications, including the use of severe caloric or protein restriction to treat diseases such as kidney failure and morbid obesity. Researchers propose considering genetic, infectious, and metabolic influences when looking for treatments, particularly in regard to HIV/AIDS.
A major gene therapy advance for treating Parkinson's disease has been funded by the Fox Foundation grant, with RheoGene Inc. developing a technology to manage gene expression. The therapy uses a patented small-molecule mediator to turn genes on or off, offering a potential solution to replacing lost dopamine in the brain.
Researchers used gene therapy to introduce a healthy copy of the dystrophin gene into mice with muscular dystrophy, repairing the entire muscle cell. The technique, developed by Michele Calos, has potential for long-term fixes for various genetic diseases.
Researchers at Memorial Sloan Kettering Cancer Center have successfully treated sickle cell disease in human models using stem cell-based gene therapy with RNA interference. The treatment corrected the production of abnormal hemoglobin and restored normal blood flow, offering new hope for patients.
Cedars-Sinai researchers have developed a new delivery system that can effectively regulate therapeutic gene expression, overcoming obstacles in bringing genetic therapies to humans. The system allows for the flexibility to turn gene expression on or off, even in the presence of an immune response, making it a critical tool for treatin...
Research confirms that individuals with a specific PTPN22 polymorphism are more likely to develop rheumatoid arthritis, with anti-CCP antibodies also playing a key role. The combination of these factors strongly predicts the onset of RA with a specificity of 100% for the disease.
Researchers at Children's Hospital of Philadelphia developed a novel technique to attach therapeutic genes to bare metal stents, reducing inflammation and restenosis. The study used animal models and found significantly lower restenosis with the new stents compared to conventional ones.
Scientists have identified 570 genes associated with Parkinson's disease, which could lead to the development of new treatments. The study analyzed brains from 23 patients and found that limiting the activity of these genes may help control or even stop the progression of the disease.
Researchers have found that Sangamo's ZFN-modified cells are resistant to HIV infection, whereas control cells are infected. The treatment works by disrupting the CCR5 gene, a receptor required for HIV entry into immune cells. This approach has advantages over other drugs in development, which require constant antagonist presence.
Researchers at Gladstone Institutes have made a breakthrough discovery about the role of miRNA-1 in the early stages of heart development. They found that miR-1 helps determine heart progenitor cells and maintain them until later embryonic stages, which could lead to new strategies for cardiac regenerative medicine.
Researchers have successfully treated hereditary spastic paraplegia by delivering a normal paraplegin protein to spinal motor neurons via gene therapy. This approach improved motor function in mice and holds promise for treating other forms of peripheral nerve damage caused by genetic mutations.