Articles tagged with Gene Therapy
Researchers developed a gene therapy strategy to treat Leber congenital amaurosis by adding copies of the normal CRX gene under its native control mechanism. This approach restored some CRX protein function and drove expression of opsins in patient-derived retinal organoids.
Researchers at Mayo Clinic have developed the first hybrid gene therapy for treating long QT syndrome, a genetic heart rhythm condition. The therapy targets the KCNQ1 gene and has shown potential therapeutic efficacy in two in vitro model systems using beating heart cells reengineered from patient blood samples.
Scientists at KAUST developed rules to protect inserted genes from nematode natural defenses, allowing for multiple generations of gene expression. A web application helps analyze DNA sequences for PATC watermarks, facilitating research on transgene silencing resistance.
Researchers have found a new type of genetic change in people with hypertrophic cardiomyopathy (HCM), which can cause sudden death. This discovery will help doctors predict which family members need to be monitored and which can be ruled out from further tests.
A phase 2 clinical trial using a personalized treatment approach found increased survival rates among patients with metastatic tumors in gastroesophageal cancers. The study used genetic profiling to tailor treatments, resulting in a 66% one-year survival rate and a median survival time of 15.7 months.
The study investigates the biodistribution of AAV gene transfer vectors in nonhuman primates using quantitative positron emission tomography (PET). The results show that both AAVrsh.10 and AAV9 vectors distribute primarily to the liver, with lesser detection in the brain after intravenous administration.
A team of researchers from Ruhr-University Bochum has developed a novel approach to treat spinal cord injuries by stimulating nerve cell regeneration with a designer cytokine. In a groundbreaking study, they successfully restored walking ability in paralyzed mice, paving the way for future human trials.
Researchers at Massachusetts General Hospital have developed a gene therapy strategy that effectively treats mice with a mutated TSC2 gene, causing the growth of noncancerous tumors. The treatment extends survival to 462 days and reduces brain damage in mice, suggesting potential for human clinical trials.
Researchers found that the HTT gene mutation affects brain and body growth, leading to increased susceptibility to brain cell death, even in children as young as six. Gene therapy trials are underway to slow disease progression and potentially prevent onset by delivering gene therapy to carriers.
Researchers found that decreased activation of gene LEF1 disrupts neuronal function and promotes hyperexcitability in brain cells, a hallmark of bipolar disorder. Increasing LEF1 expression may lead to new drug targets and biomarkers for lithium nonresponsiveness.
Antisense oligonucleotides (ASOs) target disease-causing proteins, but can affect non-targeted proteins causing side effects. Researchers have developed a DNA/DNA double-stranded oligonucleotide that enhances ASO efficacy and stability in the body.
Scientists at Tel Aviv University have developed a new gene therapy that replaces the genetic defect causing deafness in mice. The treatment enabled cells to continue functioning normally, preventing gradual deterioration of hearing.
Researchers have discovered a new way to deliver DNA-based therapies for diseases using polymers, which could lead to more affordable gene therapies or vaccines. The polymer technology works by packaging the nucleic acids in a carrier that protects them from degradation, allowing them to reach their target cells.
Researchers at the University of Exeter have identified a new treatment approach for mitochondrial diseases, such as Leigh Syndrome, by using novel drugs that metabolically reprogram mitochondria to generate energy. The study successfully normalized or improved energy production in genetically mutated microscopic worms.
Researchers Gustavo D. Aguirre, Jean Bennett, and Albert M. Maguire receive $1 million prize for developing FDA-approved gene therapy for Leber congenital amaurosis, a genetic disease causing visual impairments. Their work has enabled routine treatment of the condition, restoring vision in children and adults.
A new study identified an adenovirus gene therapy vector carrying a VEGF isoform that can improve uterine blood flow in placental insufficiency. Reduced uterine blood flow and lack of bioavailable VEGF are major causes of severe fetal growth restriction, leading to serious neonatal morbidity and death.
Researchers have discovered AAV capsid-promoter interactions in the non-human primate brain, which dictate cell-specific transgene expression. This finding has profound implications for vector design in gene therapy, challenging the traditional concept of the AAV capsid as a delivery truck.
In a groundbreaking study, gene therapy injection in one eye significantly improved vision in both eyes, with 78% of patients experiencing significant visual improvement. The treatment has shown promise in treating Leber hereditary optic neuropathy (LHON), a blinding condition affecting approximately 1 in 30,000 people.
A phase 3 clinical trial involving 37 patients showed sustainable improvements in vision after 96 weeks, suggesting the gene therapy could be a safe and effective treatment for Leber hereditary optic neuropathy. The treatment unexpectedly seemed to work in both eyes, with DNA from the vectors found in both treated and untreated eyes.
Researchers at Massachusetts General Hospital found that adding exercise to a genetic treatment for myotonic dystrophy type 1 reversed fatigue in mice. Exercise alone provided some benefit, whereas the treatment alone did not.
Scientists from Trinity College Dublin have developed a new gene therapy approach that successfully protected the visual function of mice with dysfunctional mitochondria. The treatment also improved mitochondrial performance in human cells with OPA1 gene mutations, offering hope for treating diseases like Alzheimer's and Parkinson's.
A new consortium, Accelerating Research and Innovation for Advanced Therapies (ARDAT), aims to develop standardized models for predicting ATMP immunogenicity in humans. The €25.5 million project will also build understanding of ATMP drug metabolism within a host.
A novel targeted therapy, POMHEX, has been developed to block metabolic pathways in brain cancer cells with specific genetic defects. The study found that the small-molecule enolase inhibitor effectively killed brain cancer cells missing ENO1, and showed promise in animal models of this type of cancer.
A case of in-stent thrombosis (IST) occurred in an 81-year-old man undergoing coronary bypass grafting, highlighting the complexity of antiplatelet therapy choice and genetic testing in IST risk evaluation. The patient experienced IST after converting from ticagrelor to clopidogrel therapy.
Researchers identified a non-hereditary mutation in blood cells from a patient with GATA2 deficiency that may have prevented bone marrow failure and other clinical manifestations. The mutation acted as a kind of natural gene therapy, protecting the patient from developing typical symptoms.
The NIH Platform Vector Gene Therapy (PaVe-GT) project uses AAV9 as a platform vector to develop gene therapy products for four rare diseases. The project aims to improve the delivery of therapeutic genes into target cells, paving the way for access to gene therapy for patients with difficult-to-treat conditions.
Researchers have developed a novel neuroregenerative gene therapy that converts glial cells back into functional neurons, reversing glial scar tissue. This treatment also promotes brain recovery in mouse models of ischemic stroke and Huntington's disease, offering a promising solution to the glial scar problem.
A study published on Frontiers in Cell and Developmental Biology found successful neural regeneration from brain internal glial cells for stroke repair in rhesus macaque monkeys. This gene therapy approach regenerates new neurons while also protecting injured neurons from secondary damage.
Recent studies report increased risk of rAAV mobilization in gene therapy, raising concerns for treated individuals and unintended populations. The research highlights the potential for rAAV vector production to replicate under certain conditions.
Researchers have developed a safer, more targeted way to deliver CRISPR gene therapy using light-activated liposomes. The new method uses spherical nanostructures of fat molecules to carry CRISPR molecules to specific sites in the body.
Researchers have developed a new approach to prevent toxicity seen in sensory neurons of dorsal root ganglia after gene therapy to treat neurological disorders. The approach involves modifying a transgene with a microRNA target, which reduces transgene expression and eliminates toxicity.
A new gene therapy project at the Netherlands Institute for Neuroscience seeks to repair brain damage caused by MS by identifying molecules that stimulate myelin and axon repair. The goal is to develop a drug promoting functional recovery of the nervous system, potentially revolutionizing treatment for advanced MS.
Researchers found that antiangiogenic therapy can increase the invasiveness of kidney tumors and induce a poor prognosis. A new biomarker, ALDH1A3, has been identified to predict patient response to treatment.
Researchers create genetic sensors that can detect gene activity, not just presence, using CRISPR-Cas13 system. This innovation has potential for biotech applications, including therapeutics and diagnostics.
Researchers at Yale University screened hundreds of millions of cells exposed to COVID-19 and MERS viruses, identifying dozens of genes that enable or inhibit viral replication. The study found pro-viral and anti-viral genes, which may help predict severe illness and inform treatment development.
Researchers develop reproducible protocol to assess immune effects of nucleic acid nanoparticles, a promising area of biomedical treatment. The protocol measures both quantitative and qualitative aspects of the immune response, enabling more accurate predictions and potential therapeutic uses.
A new study examines the risk of acute kidney injury among Black individuals with sickle cell trait and disease, finding a higher risk for acute kidney injury and faster loss of kidney function. The study adds important information to sparse data on the risk of acute kidney injury in those carrying the sickle cell gene.
A new study by UCLA researchers found that antiretroviral therapy given over two years was unable to completely restore age-appropriate epigenetic patterns in HIV-infected adults. This suggests that even successfully treated HIV-infected individuals are at an increased risk for early development of diseases commonly associated with aging.
A new gene therapy using a novel light-sensing protein has restored significant retinal function and vision in blind mice. The therapy involves attaching the MCO1 opsin to retina bipolar cells using gene therapy, allowing treated mice to navigate mazes and detect changes in motion faster than untreated mice.
Researchers at Mayo Clinic have developed a potential test for Machado-Joseph disease, a rare neurodegenerative disorder with no cure. The new test can quantify mutant protein levels in human biofluids, which may help evaluate the effectiveness of therapies aimed at reducing protein accumulation.
A UCI-led study demonstrates the therapeutic potential of base editing for treating inherited ocular diseases, restoring visual function to near-normal levels. The new CRISPR technology overcomes previous barriers, enabling precise and predictable correction of point mutations.
A new study has identified three gene variants that may help predict which children with eosinophilic esophagitis (EoE) are likely to respond to proton pump inhibitor (PPI) medication therapy. The research found that children carrying these variants are at increased risk of relapse after one year of PPI maintenance therapy.
Researchers create an artificial C-to-U conversion system using APOBEC1, allowing for the restoration of mutated genes and potentially treating genetic disorders. The system was tested on blue fluorescent protein (BFP) RNA with a 199T>C mutation, showing high editing efficiency.
University of Texas at Dallas researchers have identified the source of fluctuations in gene expression between identical cells, finding that transcription is the primary driver of cellular noise. This discovery has significant implications for developing targeted therapies and gene therapies.
Researchers at Kanazawa University discovered that thymoquinone selectively kills prostate cancer cells with a SUCLA2 gene deletion. This finding offers a promising therapeutic option for advanced prostate cancer patients who have developed resistance to hormone therapy.
A young woman with kaposiform lymphangiomatosis was treated with a MEK inhibitor, resulting in complete resolution of symptoms and remodeling of her lymphatic system within months. The treatment showed promise for other patients with lymphatic disorders.
Research reveals genetic differences in fat storage and formation affecting disease susceptibility in men and women. Six genes were identified as potential therapeutic targets for distinct outcomes in each sex.
Researchers identified 182 genes that regulate interaction between cancer and immune T cells, which could be harnessed for effective immunotherapy across different tumour types. The study also revealed the need for new therapy to account for genetic mutations in cancer cells that can make disease worse in response to treatment.
Researchers successfully used gene therapy to overcome cardiac effects of Friedreich's ataxia in a mouse model, achieving exercise performance similar to healthy littermates. The treatment delivered the frataxin gene via adeno-associated virus (AAV) and showed promising results.
Scientists accelerated vision restoration by visualizing photoreceptors' response to light in the living eye. Researchers modified OCT technology to detect minute changes, enabling high-resolution visualization of cone photoreceptors and potential therapies like stem cells or gene therapy.
Researchers at Children's Medical Research Institute have discovered a reason behind the low success rate of gene therapy targeting liver diseases using Adeno-associated virus 2 (AAV2). The team found that AAV2 binds too tightly to its attachment receptor, heparan sulfate proteoglycans, which leads to the vector getting
Capmatinib shows high response rate (68%) in first-line treatment for NSCLC patients with MET exon 14 skipping mutations. Patients with lower MET amplification levels have limited effectiveness with the drug.
Researchers have found that specific DNA variants can affect the response to interferon-alpha-based treatments, leading to personalized therapy options. The discovery may improve treatment outcomes for patients with polycythaemia vera, a rare chronic malignant blood disease.
A new trial suggests that children with asthma may benefit from personalized treatment based on genetic differences. The study found that children with two copies of a specific gene variant experienced an average improvement in quality of life score of 0.42.
Researchers created a system that uses CRISPR to briefly suppress genes related to AAV antibody production, allowing the virus to deliver its cargo unimpeded. The study shows promise for improving gene therapy's effectiveness and preventing or treating sepsis in mice.
A meta-analysis of NHP studies reveals that AAV gene therapy often causes DRG pathology with no clinical effects, prompting preclinical safety evaluations before clinical trials. The study's findings have the potential to streamline the development process for new vectors.
Researchers have developed a novel gene therapy approach that activates genes with similar functions to compensate for the primary defect in retinitis pigmentosa. This method has shown promise in slowing down retinal degeneration and improving retinal function without side effects.
A study by University of Pennsylvania researchers describes an effective gene therapy for Krabbe disease in dogs, which has shown promising results with no significant symptoms after four years. The treatment approach, using the AAV9 vector, has also been found to positively affect both central and peripheral nervous systems.
A new study published in Pediatrics reports positive safety and early outcome data from 21 children with spinal muscular atrophy treated with gene therapy. The treatment, onasemnogene abeparvovec-xioi, is shown to be effective through age 2 years with proper screening and monitoring, but requires closer attention for potential liver is...
Researchers have discovered new genes that play a crucial role in determining the success of radiotherapy in cancer treatment. These genes, which encode for repair mechanisms used by cancer cells, can be missing or dysfunctional in some patients, making them more likely to respond to radiation therapy.