Articles tagged with Gene Therapy
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.
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.
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.
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.
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 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.
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.
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.
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.
A gene therapy case report published in Human Gene Therapy reveals a safe and effective treatment for lipoprotein lipase deficiency, with improved quality of life outcomes after discontinuing plasmapheresis. The treatment, ali-pogene tiparvovec, has been approved in Europe to treat the rare inherited disorder.
A study by Dana-Farber Cancer Institute and Broad Institute identified five genetic subtypes of diffuse large B cell lymphoma, which can help predict individual patient outcomes and guide personalized treatment. The analysis revealed clear links between specific genetic signatures and how patients respond to standard treatment.
Researchers have identified a therapeutic RNA molecule that corrects the genetic processing error causing familial dysautonomia by enabling human and mouse cells to produce normal levels of IKAP protein. The discovery provides proof of concept for potentially treating this rare disorder.
Researchers have developed a gene therapy approach to treat GM2 gangliosidosis, a devastating neurodegenerative disease. The treatment has shown promising results in mouse models and is now being tested in clinical trials using recombinant Adeno-associated viruses for gene transfer.
A global research project has identified 44 genetic variants linked to major depression, 30 of which are newly discovered. These findings suggest a shared genetic basis with other psychiatric disorders like schizophrenia.
Researchers at Duke University successfully delivered CRISPR/Cas9 repressors to silence the Pcsk9 gene, which regulates cholesterol levels, in adult mice. The treatment resulted in reduced blood cholesterol levels and sustained gene repression for six months after a single treatment.
A novel technique integrates CRISPR technology with big data mining to identify functional roles of lncRNAs in chemotherapy resistance. The approach identified hundreds of new genetic units impacting sensitivity to chemotherapy, providing potential biomarkers for diagnosis and treatment.
A new gene therapy has successfully treated transfusion-dependent thalassemia, a blood disorder that requires frequent red blood cell transfusions. The treatment produced positive outcomes in an interim analysis of two international clinical trials, with most patients becoming transfusion-free.
A gene therapy treatment for cerebral adrenoleukodystrophy (ALD), a fatal degenerative brain disease, has been successfully tested in a clinical trial. The treatment halted the progression of the disease in 15 out of 17 boys who participated in the trial, leading to minimal or no symptoms and normal lives.
A new gene therapy approach has been shown to reduce the need for blood transfusions in patients with severe beta-thalassemia. The treatment, known as LentiGlobin BB305 vector, was effective in eliminating the need for transfusions in 73% of patients, paving the way for potential treatment.
A new liquid biopsy-based cancer model has revealed insights into the development of chemotherapy resistance in small-cell lung cancer. The study found that intratumoral heterogeneity, or differences in gene expression between tumor cells, contributes to rapid chemotherapy resistance.
Researchers developed a powerful mouse model using CRISPR-Cas9 and RCAS/TVA system to mimic glioma genetic alterations. The model enables the study of gene fusions and mutations in cancer progression and treatment response.
Researchers engineered a donor cornea with genes that inhibit vascularization, reducing the risk of tissue rejection in high-risk patients. The study shows promising results for gene therapy in preventing corneal graft rejection.
Researchers used machine learning to classify abnormal protein activity in tumors, identifying potential 'hidden responders' who may benefit from specific therapies. The study combined genetic data with machine learning approaches to predict response to inhibitors affecting cancer cells with overactive Ras signaling.
Scientists have developed a method to efficiently genetically modify T-cells from mice, allowing for targeted gene surgery and potential therapeutic applications. The technique involves using plasmids and CRISPR-Cas9 molecular scissors to introduce specific changes into the cells.
Researchers discovered a parasite-derived protein, H-IPSE, with potent therapeutic effects on chemotherapy-induced hemorrhagic cystitis in mice. The study found that H-IPSE alters host cell transcription, suggesting a novel approach to treating bladder pain and injury.
Researchers developed novel strategies to engineer more stable and active proteins, enhancing mRNA therapy potential. Sequence engineering improved protein expression, duration, and enzymatic activity in mice.