Articles tagged with Gene Therapy
Researchers describe a DNA immunization platform that stimulates innate and adaptive immunity to produce high levels of antigen-specific antibody responses. The approach activates multiple pathways of innate immunity, leading to the development of high-affinity antibodies in mouse, rabbit, and human models.
Researchers developed a novel DNA vaccine that protected mice against multiple H3N2 viruses and severe illness, offering a promising approach to elicit an immune response against diverse strains. The vaccine strategy represents a step forward in eliminating the need for seasonal reformulation of influenza vaccines.
Researchers at the University of Pennsylvania School of Medicine have discovered that viral vectors used in gene therapy undergo spontaneous changes during manufacturing, affecting their structure and function. The team has developed new ways to prevent these changes, leading to more efficient and safer delivery of gene therapies.
Warren Ruder, a Pitt engineer, has been awarded $1.5 million from the NIH Director's New Innovator Award to develop magnetically induced synthetic gene networks for cell and tissue therapies. His research combines biology and engineering to create new biomimetic systems that can regulate disease pathways.
A study comparing DNA and RNA data from Nigerian breast cancer patients to those in a US database found aggressive molecular features prevalent in tumors from Nigerian women, which could explain disparities in breast cancer mortality. The study highlights the potential for targeted therapies to reduce mortality gap.
MIT biological engineers have developed a way to regulate RNA expression, giving precise control over the dose of therapeutic protein a patient receives. This technology allows doctors to tailor treatment for individual patients and offers a quick way to turn off protein production if necessary.
A multidisciplinary team of scientists from the University of Pennsylvania, Children's Hospital of Philadelphia, and University of Wisconsin-Madison are developing a stem cell-based approach to treat blindness in dogs. The researchers aim to generate specialized retinal cells using adult stem cells and transplant them into the retina.
Researchers have developed a two-pronged approach targeting Ebola virus infection using linked nucleic acid (LNA) antisense oligonucleotides (ASOs). The study demonstrates effective targeting of two Ebola viral genes and the ability to prevent infectivity via Niemann-Pick C1.
Researchers used a novel method to analyze individual brain cells in mice, revealing specific genes that may play roles in traumatic brain injury. Treatments targeting these genes, such as thyroid hormone therapy, showed promise in improving learning and memory tasks.
Researchers at Johns Hopkins Medicine used an endoscopic procedure to deliver therapeutic genes to the liver via the common bile duct, demonstrating a safe and effective method for gene therapy. The technique, called ERCP, was found to be less invasive than traditional injection methods, with no biliary or liver injury observed.
Kathryn Whitehead's project uses genetic engineering to isolate and modify human cells in breast milk for infant disease therapy. The goal is to develop non-invasive treatments for babies with diseases, such as spina bifida or enterocolitis.
Researchers discovered that driver gene mutations are remarkably similar across different metastases from the same patient, offering hope for successful targeted therapies. This finding suggests that single biopsies can capture essential information for therapeutic decision-making.
Researchers developed a genome editing tool to target and eliminate damaged mitochondrial DNA, resulting in improved mitochondrial function in mice. The treatment, known as mtZFN, could provide a practical route to treating patients with mitochondrial diseases.
The RAC's elimination would shift review responsibility to local Institutional Biosafety Committees. The new approach aims to reduce redundancies between FDA and RAC roles in human gene therapy.
A promising gene therapy for visually impaired sheep has been approved for human trials, offering hope for patients with achromatopsia. The treatment has already shown success in treating affected sheep and demonstrates the potential for a cure.
A study led by the University of Exeter Medical School found that disrupting genes and pathways regulating splicing factors can reverse signs of aging in cells. Disrupting ERK and AKT pathways reduced senescent cells, increasing splicing factors and leading to cellular rejuvenation.
Researchers at the Salk Institute and University of Florida used cryo-electron microscopy to image an AAV2 virus with improved resolution. The study reveals key changes in the viral portal that enable it to be less infectious, potentially informing gene therapy applications for various diseases.
A team of researchers has developed a gene therapy cure for the Leber Congenital Amaurosis, a genetic form of childhood blindness. The treatment restores vision to treated children and adults, paving the way for new treatments for genetic conditions.
Researchers at Stanford Medicine have successfully developed a method to induce tolerance to gene therapy in mice, eliminating the autoimmune reaction that often occurs. This breakthrough could lead to effective treatments for single-gene disorders such as Duchenne muscular dystrophy.
Researchers at Gladstone Institutes identify 156 genes affecting ATP levels in cells, shedding light on cellular energy production. The findings could lead to the development of new therapies for diseases such as mitochondrial disorders, heart disease, and neurodegenerative diseases.
Studies with rodent models show reduced anxiety and improved brain function with early treatment using the histone deacetylase inhibitor LBH589. This approach may offer a new therapeutic strategy for Huntington disease.
A recent study has identified 24 genes that are differentially expressed in rats with cerebral aneurysms compared to those without. These genes may enable the development of diagnostic biomarker tests for patients at increased risk of cerebral aneurysm formation.
The NIH grant will support research on biological treatments and clinical diagnosis for patients with hearing loss, a condition affecting over 28 million Americans. Xue Zhong Liu's team will expand a genomic database and assess the impact of genomic testing on patients.
Gene therapy using optimized AAV to deliver human factor VIII showed substantial hFVIII expression and no detectable antibody response in cynomolgus macaques. The results suggest that AAVhu37-based gene therapy has the potential to advance to clinical trials for treating hemophilia A.
Researchers developed a gene therapy that eliminates the abnormal copy of rhodopsin and restores it with a healthy copy, preserving retina's light-sensing photoreceptor cells. This approach has the potential to treat a large percentage of patients with rhodopsin autosomal dominant retinitis pigmentosa.
Researchers developed a single gene therapy vector that preserved retinal structure and function for over 8 months in a canine model of autosomal dominant retinitis pigmentosa. The treatment had potential implications for treating inherited retinal degeneration associated with the rhodopsin gene.
Scientists have developed a novel gene therapy that effectively reduces rhodopsin production and prevents photoreceptor death in dogs with autosomal dominant retinitis pigmentosa. The treatment, which combines shRNA interference with a replacement gene, shows promise for slowing or preventing vision loss in humans.
Researchers at CNIO develop gene therapy with telomerase, proving effective in mice against diseases caused by excessive telomere shortening. The study finds that the gene therapy does not increase the risk of developing cancer, even in a cancer-prone setting.
Scientists have identified nearly 200 potential drug targets for tuberculosis, a complex disease caused by bacteria made up of 4,000 genes. The research found that specific human proteins, such as CBL, can limit bacterial infections and potentially provide a new approach to combat the disease.
A study found significant improvement in cone function and no abnormalities in treated eyes for up to six years. The treatment used an AAV5 vector delivered a CNGA3 gene therapy, offering hope for treating genetic blindness.
A new study shows that a single injection of AAVB1-GAA gene therapy prolonged survival and improved enzyme activity in a mouse model of Pompe disease. The therapy also targeted the respiratory system, improving ventilatory measures.
A new natural history study of Amish nemaline myopathy provides a platform for exploring gene replacement therapy. The study's findings show promise for treating the lethal disorder, which is linked to a mutation of the TNNT1 gene.
A study by University of Utah Health scientists reveals that variation in the genetic modifier Baldspot can influence disease severity in retinitis pigmentosa. The research suggests targeting genes like Baldspot could lead to effective treatments against some disorders.
A new article explores the role of inflammatory cytokines in creating T cell exhaustion in cancer, suggesting a direct pathway for therapeutic interventions. The study found that reprogramming exhausted T cells and anti-inflammation therapy may improve antitumor activity.
Jackson Laboratory Assistant Professor Mingyang Lu has developed a computational approach called RACIPE to capture genetic events in cellular decision-making. The algorithm could have broad impact on basic research and designing new therapeutic interventions in genomic medicine.
The Alliance for Cancer Gene Therapy (ACGT) is funding innovative clinical approaches to find gene therapy treatments for solid cancers. Recipients of the 2018 research grants include a team working on a vaccine for melanoma, an injectable genetic agent to program T cell receptors, and CAR T cell therapy for brain cancer.
Chinese researchers have improved the adenine base editing (ABE) system to generate mouse or rat models for human genetic disorders, such as DMD and Pompe disease. The ABE system allows for efficient alteration of genetic codes with minimal undesired outcomes, making it a promising technology for therapeutic applications.
Researchers developed a gene therapy that restored function to the olfactory system in genetically modified mice. The therapy increased cilia and sensory neuron responses, leading to restored connections between neurons and behaviors in odor-guided tasks.
Researchers at OHSU have discovered a naturally occurring neurodegenerative disease in monkeys that mimics Batten disease, a fatal childhood disorder. The study confirms the CLN7 gene mutation and provides a promising model for developing new gene therapies.
A new study published in Nature Medicine found that fetal gene therapy can prevent and cure neonatal lethal neurodegenerative diseases in humans in utero. The therapy was tested on mice with Gaucher disease and showed promising results, including improved brain degeneration and increased survival time. Researchers believe this approach...
Researchers from UAB successfully used gene therapy to treat type 2 diabetes and obesity in mice by inducing the production of FGF21, a hormone that maintains correct energy metabolism. The treatment led to weight loss and improved insulin sensitivity, reducing the risk of related comorbidities.
Researchers have successfully used viral vectors to deliver genetic material to kidney cells, paving the way for new treatments for chronic kidney disease. The discovery could lead to gene therapies that slow or reverse the growth of cells contributing to CKD.
Researchers at Washington University School of Medicine have developed a gene therapy method to target damaged kidney cells, which could lead to improved treatment for chronic kidney disease. The approach uses adeno-associated virus (AAV) to deliver genetic material to targeted cells, showing promise in slowing or reversing cell damage.
Researchers successfully used CRISPR-Cas9 genome editing to restore normal levels of the AAT enzyme in mouse models of alpha-1 antitrypsin deficiency. The approach showed promise for treating patients with this genetic disorder.
A new molecular imaging method allows early assessment of gene therapy success, potentially improving treatment for Parkinson's and Alzheimer's diseases. The PET reporter gene/probe system enables noninvasive monitoring of gene expression in all brain areas.
Cystic fibrosis patients may benefit from a new lipid-based nanoparticle therapy that enables cells to produce the correct protein, allowing for improved respiratory function. The therapy has shown promise in treating all cystic fibrosis patients, regardless of their genetic mutation.
Researchers found substantial overlaps in genetic risk for psychiatric disorders, including ADHD, bipolar disorder, major depressive disorder, and schizophrenia. In contrast, neurological disorders showed limited evidence of common genetic risk factors, suggesting greater diagnostic specificity.
University of Michigan researchers have made a breakthrough in treating Spinocerebellar ataxia type 3 (SCA3) using gene silencing technology. They successfully reduced mutant RNA levels without toxic effects, offering hope for disease-slowing therapy.
A new RNA-Seq assay detects gene fusions in solid tumor cells with high accuracy, identifying 93% of fusions missed by other techniques. The assay also discovers 18 novel fusions, including 11 previously unknown combinations, which may have clinical significance.
AcuraStem has been awarded a $3.7 million SBIR grant to continue research on a small molecule therapeutic for ALS, utilizing induced motor neuron cellular models and precision platform iNeuroRx°. The grant supports the development of AS2015, targeting expansion repeats in the C9ORF72 gene.
A novel medication, BYL719, demonstrates efficacy in treating CLOVES Syndrome by reducing vascular masses and improving quality of life. Patients show significant symptom reduction, growth stabilization, and improved overall health.
A rapid test can distinguish infants with a specific genetic change from those without it, allowing tailored prescribing and preventing antibiotic-related deafness. The test uses a cheek swab and can produce results in around 40 minutes.
A new gene therapy has been shown to restore hand function in rats with spinal cord injuries by breaking down scar tissue and allowing nerve cell regeneration. The therapy uses a 'stealth gene' switch that can be turned on and off, providing a safeguard and paving the way for human trials.
The study found that matching targeted therapies to tumor-specific gene mutations improved progression-free and overall survival in patients with advanced disease. Receiving matched targeted therapy was also an independent factor for predicting longer overall survival.
A novel data-driven approach called PanDrugs prioritizes cancer treatments based on patient genetic alterations. The method integrates pathway context and collective gene impact to provide therapeutic options for patients with limited druggable genes.
Researchers at Kyoto University developed a synthetic molecular code that can script gene activation, targeting histones and emulating the natural histone acetylation process. The code, called Bi-PIP, successfully activated a specific gene associated with central nervous system disorders in living cells.
Dr. Michel Sadelain shares his role in advancing CAR T-cell therapy, a rapidly evolving field that targets cancer cells. He describes the FDA approval of the first CAR T-cell therapy and predicts next-generation technology advancements.
Researchers at Michigan State University have developed a gene therapy that restored vision in dogs with progressive retinal atrophy, a condition similar to human retinitis pigmentosa. The treatment has shown a 100% success rate and is now being advanced for human use with a $8.2 million grant.
The Damon Runyon Cancer Research Foundation has announced five new Physician-Scientist Training Awards, which will provide $2.3 million in funding for the recipients. The awards aim to address the financial disincentives that often deter physicians from pursuing a research career.
Tivanisiran, an RNAi-based compound, shows promise in treating dry eye syndrome by improving ocular inflammatory parameters and reducing symptoms. The Phase III Helix study will evaluate its efficacy in over 300 patients across six countries.