Articles tagged with Gene Therapy
Researchers identified key risk genes associated with schizophrenia and their impact on antipsychotic medication efficacy. Examining these single nucleotide polymorphisms (SNPs) can help differentiate between responsive and treatment-resistant patients.
A Canadian pilot study on gene therapy for Fabry disease shows the treatment is working and safe, enabling patients to produce normal levels of the defective enzyme. The trial, led by Dr. Aneal Khan, treated five men with a single dose of gene therapy, which resulted in stable patients who are no longer requiring replacement therapy.
Researchers found that genes affecting cilia function are linked to diabetes, kidney failure, and liver fibrosis in both rare genetic disorders and the general public. The discovery opens up new possibilities for targeted treatments and gene therapies.
Researchers have developed an immune tolerance platform called ImmTORTM to overcome challenges in gene therapy, including immunogenicity and durability. The addition of ImmTOR nanoparticles to AAV vectors has shown potential to enhance efficacy, safety, and durability by mediating more efficient transgene expression.
Researchers have developed improved gene vectors for ocular gene therapy, allowing for widespread delivery and reducing risks associated with traditional approaches. These vectors are being tested in clinical trials and have shown promise in restoring daylight vision in animal models of achromatopsia.
Researchers develop DNA modifying epigenetic therapy to transform immune killer T-cells into "super soldiers" with enhanced ability to kill cancer cells. The therapy uses available chemotherapy drugs to remove epigenetic tags, turning key genes back on and turbocharging the cells' killing function.
A new biomarker has been identified that can determine individual treatment duration for tuberculosis patients, reducing the need for prolonged therapy. The biomarker is based on an RNA signature from 22 genes and was developed using patient cohorts from Germany and Romania.
Researchers from Indiana University have identified key genetic changes in the interstitial kidney tissue of people with diabetes, which could lead to a revolutionary new genetic approach to treating kidney disease. The study found that important genes change when a scar forms on the interstitium, a previously undercharacterized part o...
Researchers found higher than expected genetic changes in a group of 100 MND patients, suggesting routine genetic testing may be beneficial. The study recommends genetic testing for all MND patients, regardless of family history, to improve disease subclassification and tailored treatments.
Researchers used artificial intelligence to generate a large library of distinct AAV capsid variants, achieving a 60% viable yield. This approach overcomes the limitation of current vectors and expands the number of diseases treatable with gene therapies.
Researchers at Wyss Institute and Google Research used machine learning to design highly diverse AAV capsid variants that can evade neutralizing antibodies. The approach produced over 57,000 variants with improved functional diversity, potentially leading to improved gene therapies and reduced immunogenicity.
A new AAV-delivered gene therapy strategy uses immunomodulation to reduce inflammation and improve efficacy. By incorporating TLR9-inhibitory sequences into the AAV genome, researchers have enhanced expression of transgenes in mice, suggesting potential for higher efficacy.
Researchers have developed targeted ultrasound techniques to treat various brain diseases such as Alzheimer's, Parkinson's, and stroke. The new methods aim to improve brain functions by activating functional neurons without significant side effects.
The Canadian Neuromuscular Disease Registry has facilitated over 125 research projects and secured funding for patients across Canada. With data from over 4,000 patients, the registry provides valuable insights into neuromuscular disease outcomes and care.
Researchers discovered that three patients with DOCK8 deficiency spontaneously repaired their faulty genes through somatic reversion, restoring normal immune function. This breakthrough has implications for future therapies and treatments for the often-fatal disease.
The AAVCOVID vaccine program has received a $2.1 million grant to support phase I clinical trials overseas, with single-dose vaccines that can be stored at room temperature for up to one month. This stability could enable distribution in regions with limited infrastructure.
Researchers at Children's Hospital of Philadelphia developed a new gene therapy vector that produces more hemoglobin with a lower dose, minimizing toxic side effects. The vector, ALS20, was found to be significantly more effective than current vectors in treating beta-globinopathies.
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 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 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.
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.
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.
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 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 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.
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 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.
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.
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.
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.
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 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 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 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.
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 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.
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.
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.
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.
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 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 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 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.