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 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.
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 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.
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.
Researchers at Cedars-Sinai have developed a novel gene therapy that uses immune cells to target glioblastoma multiforme. A specific biomarker, HMGB1, has been identified as an effective tool to monitor tumor response to this treatment.
Scientists have made significant discoveries in the fight against muscular dystrophy, identifying a potential gene therapy approach to overexpress Galgt2 protein. In another breakthrough, researchers have identified a stromal marker for breast cancer progression, highlighting the importance of examining stroma PDGF receptor expression ...
A TGen-led team has identified five genetic biomarkers that may predict response to the anti-diabetes drug Actos, enabling personalized medicine for patients with type 2 diabetes. These markers were found in genes associated with PPARG function and include variants in a key drug metabolizing gene called cytochrome P450 3A4.
Researchers have developed a gene delivery approach to target therapeutic genes to nerves in the dorsal root ganglion (DRG), a region affected in various sensory neuronopathies. This method, using helper-dependent adenoviruses, was found to be more efficient at delivering genes to DRG nerves compared to nontargeted versions.
Researchers discovered that delivering a small molecule that is highly expressed in normal tissues but lost in diseased cells can result in tumor suppression. The study employed a novel strategy to treat hepatocellular (liver) cancer, using microRNAs (miRNA) gene delivery via a recombinant adeno-associated virus (AAV).
Researchers develop synthetic protein that induces blood clotting, cutting off tumor's nutrient and oxygen supply. The treatment resulted in a 53% decrease in tumor volume and slowed cancer cell growth by 49%.
Researchers develop gene therapy approach to target Factor VIII in hemophilia A mice, reducing disease symptoms. L-Asparaginase degradation mechanism also uncovered, with potential for optimizing treatment.
Researchers at the University of Minnesota Medical School have successfully provided long-term expression of Factor VIII in hemophilia A mice using a new gene therapy approach, marking a promising step toward human clinical trials.
A team of researchers identified astrocyte elevated gene-1 (AEG-1) as a critical gene that regulates tumor progression in neuroblastoma, a form of cancer commonly found in young children. Loss of AEG-1 was shown to reduce the tumor-causing properties of aggressive neuroblastoma cells.
A team of researchers has successfully developed a combined stem cell-gene therapy approach that cures human Fanconi anemia, a genetic disorder causing bone marrow failure and cancer. The treatment uses induced pluripotent stem cells to differentiate into healthy blood cells, offering a potential cure for the disease.
Researchers at UF have developed gene therapy that restores the faulty enzyme for glycogen storage disease type 1A, allowing dogs to survive for extended periods without glucose supplementation. The treated dog, born with the deadly disease, is now 20 months old and healthy after receiving precise doses of cornstarch every few hours.
Researchers have discovered a new therapy that substitutes the missing protein dystrophin to repair weakened muscle tissue in mouse models with Duchenne muscular dystrophy. The TAT-utrophin-based protein approach addresses the cause of the disease without gene replacement or stem cell issues, offering a potential treatment option.
A study by University of Florida researchers suggests that the nervous system may be the primary cause of severe breathing problems in children with Pompe disease, a rare genetic disorder. The discovery shifts responsibility from muscle weakness to signaling between the spinal cord and the diaphragm.
Researchers discuss a combined approach to maximize the benefits of stem cell therapy while minimizing risks. Gene therapy has the potential to solve the problem of wayward stem cells wreaking havoc after transplantation by genetically modifying them prior to use.
INTUNIV demonstrated significant efficacy in treating ADHD symptoms, particularly in children aged 6-12 years with oppositional behaviors. The study showed improved symptom reduction on various rating scales, including the ADHD Rating Scale-IV and Clinical Global Impressions-Improvement.
Researchers at Fox Chase Cancer Center have discovered a genetic pattern that can help predict how patients with gastrointestinal stromal tumors respond to the targeted therapy imatinib mesylate. By analyzing tumor specimens, they found genes that could be suppressed to enhance the effectiveness of imatinib therapy.
Researchers have identified a genetic signature that can predict which breast cancer patients will respond well to treatment with epirubicin, a widely used form of chemotherapy. This signature may help ensure fewer women are exposed to severe side effects by identifying those most likely to benefit from treatment.