Articles tagged with Gene Therapy
A second dose of gene therapy has proven safe and effective in animal studies, suggesting that patients who benefit from treatment in one eye may experience similar benefits in the other. The study found no signs of toxic side effects or interference from unwanted immune reactions.
Researchers used gene therapy to deliver a portion of DNA that makes the SMN protein into newborn mice, resulting in improved muscle coordination and longer survival. The treatment corrected motor function, restored nerve signals, and improved overall health in mice with spinal muscular atrophy.
Fetal surgery continues to evolve with advancements in prenatal diagnosis and surgical techniques. Experts at the Children's Hospital of Philadelphia describe innovative procedures for treating rare defects, including lung masses, sacrococcygeal teratomas, and spina bifida.
Physicist Alex Evilevitch directly measured the energy associated with viral DNA expulsion, a discovery that could lead to broad-spectrum antiviral drugs. The study used isothermal titration calorimetry and found that increasing DNA length increases heat release, highlighting the importance of hydration entropy in viral genome packaging.
A new study by the University of Pennsylvania School of Medicine found that extended use of a nicotine patch improves abstinence from cigarettes and helps smokers quit again after relapse. The extended therapy regimen was more effective in boosting abstinence rates than standard therapy.
A new tool called PEG-POD significantly increases gene delivery to retinal cells, outperforming existing carriers and DNA alone. This breakthrough may help develop therapies for conditions like retinitis pigmentosa and age-related macular degeneration.
University of Florida researchers are developing a gene therapy to treat Pompe disease, which causes severe muscle weakness and breathing difficulties. The treatment involves delivering a corrective gene directly to the diaphragm using an adeno-associated virus, with clinical trials expected to begin soon.
Researchers have achieved a breakthrough in regulating genes by hijacking riboswitches, opening doors for targeted drug discovery and synthetic biology. The study, published in Proceedings of the National Academy of Sciences, uses synthetic molecules to activate genes previously controlled by small naturally occurring molecules.
Researchers have identified a human genetic alteration associated with anxiety-related behavior in both humans and mice, which can inform the development of new clinical strategies to treat anxiety disorders. The study used functional magnetic resonance imaging (fMRI) to reveal altered brain function in individuals with this abnormality.
Researchers found that gene therapy to express a constitutively active form of protein I-1c in heart muscle cells improved contractile function in young mice, but led to abnormal heartbeats and sudden death under stress. Older mice developed characteristic features of heart failure after treatment.
Neuroscientists combined herpes and Sleeping Beauty to deliver larger genes to brain cells, increasing the size of genetic payload by nearly tripling it. This breakthrough technology could lead to treatments for devastating brain diseases like pediatric leukodystrophies and lysosomal storage disorders.
A study published in Cancer Cell reveals that epigenetic differences can distinguish patients with acute myeloid leukemia (AML) into subtypes with varying responsiveness to therapy. A set of 15-gene DNA methylation biomarkers was found to be highly predictive of patient survival.
Researchers from Boston University School of Medicine have discovered a gene therapy that can prevent the progression of emphysema by delivering therapeutic genes to lung tissue. The treatment achieved sustained expression of human alpha-1 antitrypsin protein, improving lung compliance and alveolar size in mice with emphysema.
An international team of researchers has identified a gene associated with DFN2, a rare form of progressive deafness that primarily affects males. The discovery offers therapeutic implications and may lead to treatments for other types of deafness.
Researchers identified specific genetic changes associated with an increased risk of relapse in cervical cancer patients. These changes are crucial steps in the progression of the disease towards an aggressive and treatment-resistant state.
Researchers at German Cancer Research Center find that HIV virus rarely integrates into genes with high transcription activity. This discovery could lead to the development of specific AIDS drugs targeting these 'cold spots', potentially offering a new approach to combat the virus.
Researchers have successfully transplanted genetically modified hematopoietic stem cells into mice, allowing their developing red blood cells to produce a critical lysosomal enzyme and preventing or reducing organ and central nervous system damage from Hurler's syndrome. This approach has the potential to improve treatment options for ...
Researchers have achieved significant success with gene therapy in treating adrenoleukodystrophy (ALD), a potentially fatal brain disorder. The treatment has arrested the progression of the disease in two boys, showing no adverse effects to date.
Researchers have successfully used a gene therapy technique to slow the onset of X-linked adrenoleukodystrophy, a severe brain disease, in two patients. The treatment involved introducing a therapeutic gene into blood cells using a lentiviral vector, which showed promise in slowing disease progression and improving neurological function.
Gene therapy has been successfully tested in France, halting the progression of adrenoleukodystrophy in two children. The innovative approach uses a modified HIV virus to deliver a therapeutic gene to bone marrow cells, which then migrate to the brain and display correcting potential.
Researchers successfully used gene therapy to repair injured human donor lungs, making them suitable for transplantation. The technique improves lung function and reduces inflammation, offering a promising approach to increase organ availability and improve patient outcomes.
A gene therapy treatment has successfully restored vision in five children and seven adults with Leber's congenital amaurosis, a group of inherited blinding diseases. The treatment uses genes to produce light-sensitive pigments in the back of the eye, allowing patients to navigate low-light environments and perform daily activities.
Gene therapy has been shown to improve vision in children with Leber congenital amaurosis, a severe form of inherited retinal degeneration. The treatment resulted in significant improvements in both subjective and objective measurements of vision.
Researchers determine the three-dimensional structure of Pur-alpha protein, essential for normal neural function, and gain insights into its molecular function. The findings provide a possible basis for developing an effective therapy for Fragile X tremor/ataxia syndrome.
A new publication in Molecular Therapy outlines the dramatic effects of an exon skipping PPMO in preventing severe deterioration of dystrophin-deficient mice. The treatment restores dystrophin protein expression and maintains near-normal muscle function, suggesting great potential for treating Duchenne muscular dystrophy patients.
Researchers have tested various cell-based therapies in mice with Alport syndrome, a genetic kidney disease. Stem cell treatments may repair kidney defects associated with the disease, offering promise for patients.
Researchers have identified a genetic factor that increases the risk of sudden cardiac death in patients with congenital long-QT syndrome. Variants of the gene NOS1AP were found to predispose individuals to a worse form of the disease, leading to longer QT intervals and increased symptoms.
Cold Spring Harbor Laboratory scientists uncover a large cache of genes that act as built-in barriers against cancer, including over 10 new tumor suppressor genes. The study reveals that even partial loss of function in these genes can accelerate tumor growth.
Parkinson's Disease (PD) incidence increases with age, with a wide variation in onset age ranging from 20 to 90 years. Researchers have identified genes influencing PD onset age, which may help identify mechanisms and therapeutic targets for delaying symptoms.
Scientists used gene transfer to increase heart muscle cell contractions, improving cardiac function and quality of life for patients. The fast molecular motor gene transfer showed promise in reversing the course of heart failure.
Scientists at the University of York have successfully silenced a gene that appears essential to cancer cell survival, leaving healthy cells unaffected. This discovery suggests that certain genes may be specific to cancer cells, paving the way for the development of new cancer treatments with fewer side effects.
Researchers have discovered 231 new genes linked to head and neck cancer, which could aid in early detection and treatment. These findings may also help monitor disease progression and patient response to therapy.
Van Andel Institute researchers found the gene RhoB is crucial in the progression of Myelodysplastic Syndrome (MDS), a bone marrow disease affecting up to 15,000 people annually. The study's findings may lead to new treatments for late-stage MDS and its potential applications in leukemia and solid tumors.
Researchers are conducting a phase II gene therapy trial using CERE-110 to treat patients with mild Alzheimer's disease. The study aims to determine the safety and benefits of injecting the gene into the brain, which produces Nerve Growth Factor to help nerve cells survive.
Researchers have uncovered the genetic switch that controls macrophage polarization, essential for muscles to regenerate properly. Macrophage polarization allows them to shift from clearing debris to promoting repair in damaged areas.
Researchers at the University of Edinburgh have identified a protein that enables sections of so-called junk DNA to be cut and pasted within genetic code. This finding could speed up the development of gene therapies by allowing scientists to control the process of DNA transposition.
Researchers have found a genetic link between inherited cardiac arrhythmias and thyroid dysfunction, suggesting that mutations in the KCNE2 and KCNQ1 genes could also cause thyroid problems. The study's findings may lead to new treatments for both heart and endocrine diseases.
Researchers from the University of Florida and the University of Washington successfully used gene therapy to restore color vision in two squirrel monkeys. The study demonstrates the potential for this treatment to target adult vision disorders involving cone cells, a crucial step towards developing therapies for human cone diseases.
A Mayo Clinic study has found two genes associated with good central nervous system repair in mice with multiple sclerosis. The identification of these genes may lead to the development of new therapies and a better prediction of patient outcomes.
GS-101 demonstrates significant inhibition and regression of corneal neovascularisation in a phase II study. The treatment is generally well-tolerated with no serious side effects, offering a new approach to managing ophthalmic angiogenesis.
A new tumor marker has been identified for brain tumor resistance, allowing for targeted therapy approaches. The discovery of a
The PREDICT score identifies individual risk for poor responsiveness to clopidogrel through non-genetic factors. Studies show that response to clopidogrel is affected by genetic variants involving enzymes responsible for absorption and bio-activation of the medication.
Researchers created a new gene therapy vector that uses the same machinery as viruses to deliver therapeutic genes into cells' nuclei. This breakthrough improves efficiency and specificity of human gene therapy, paving the way for clinical applications.
Researchers have discovered a way to regulate gene therapy for Parkinson's disease by introducing an 'off switch' using the antibiotic doxycycline. This breakthrough addresses safety concerns and paves the way for safer treatment options.
A $3.5-million NIH grant will help advance a gene therapy for Tay-Sachs disease from animal tests to human clinical trials. The Boston-based consortium has successfully paired two genes in a single vector, which they believe will increase therapeutic efficiency and lower production costs.
Parkinson-associated genes PINK1 and Parkin functionally interact to maintain mitochondrial function, impairing neuron loss
MU researchers discovered a 'longevity' gene that energizes mice during exercise, potentially applicable to humans. The gene removes toxic free radicals from mitochondria, improving life quality and treating diseases like muscular dystrophy and diabetes.
Researchers found that HPRT gene plays crucial role in regulating transcription factors' expression and development of neurons, supporting a possible link to Lesch Nyhan disease. The study provides first direct experimental support for HPRT's involvement in neurological disorders.
Researchers have documented remarkable vision improvements in three young adults with Leber Congenital Amaurosis after receiving gene therapy. The patients continue to experience increased sensitivity to light, and one patient was able to read an illuminated clock for the first time at 1 year after treatment.
Researchers have discovered that gene therapy can restore vision in adults with Leber congenital amaurosis type 2, a rare form of blindness. The treated parts of the retina have acquired image-processing strength to rival the normal center for visual perception.
Researchers report significant visual benefits in three young adults with Leber congenital amaurosis, a previously untreatable form of blindness. The gene therapy, which restored the RPE65 protein necessary for vision production, has shown safety and stability over one year.
Researchers successfully delivered functional genes to patients with alpha-1 antitrypsin deficiency, triggering the production of a protective protein. The study shows promise for gene therapy as a potential treatment option for this genetic disorder.
Researchers at University of Florida have safely given new genes to patients with alpha-1 antitrypsin deficiency, enabling them to produce trace amounts of the protective protein for up to one year. This potential step toward a gene therapy could benefit approximately 100,000 Americans with the condition.
Researchers at the University of Leeds have successfully prevented epilepsy in mice by identifying a faulty gene as the cause. The study, published in PNAS, offers irrefutable proof that a defective version of the Atp1a3 gene is responsible for causing epileptic seizures in mice.
A study of nearly 1,600 tumor samples found that CT-X genes are expressed in half of ER-negative and triple-negative breast cancers. This suggests a potential new therapy approach for these aggressive breast cancer types. Researchers plan to initiate clinical trials based on the findings.
Researchers at UNC School of Medicine have found an efficient way to deliver a corrected gene to lung cells, restoring normal function in tissue models. This breakthrough could lead to improved treatment options for cystic fibrosis patients.
Researchers at University of North Carolina have discovered a new approach to delivering a corrected CF gene to lung cells using parainfluenza virus, which could potentially cure cystic fibrosis lung disease. The method has shown promise in laboratory models and may pave the way for future clinical trials.
A research team has used single-molecule fluorescence microscopy to follow magnetic nanoparticles as they transport genes or drugs into target cells in real-time. The study reveals bottlenecks in nanoferry transport and provides insights into optimizing existing systems, potentially leading to breakthroughs in gene therapy.
Researchers developed an ideal strategy for developing HCV-specific gene therapy by identifying the OAS promoter as a potential target. The study demonstrated that the HCV-core protein activates the OAS promoter specifically and effectively, paving the way for potential targeting of HCV using this approach.
A recent study published in JAMA Network has found a network of altered genes that appear to play a crucial role in the development and progression of brain tumors. The researchers identified seven landscape genes that are associated with patient prognosis and survival.