Articles tagged with Gene Therapy
The new AI model leverages hypergraphs to quickly and accurately identify therapeutic gene targets for diseases. HIT outperformed existing models in all tested metrics, demonstrating its accuracy in classifying therapeutic gene targets with great precision.
A new study reveals that radiotherapy has opposite effects on glioblastoma multiforme (GBM) and low-grade gliomas (LGG), with GBM patients living longer after treatment. The study highlights the need for personalized treatment approaches based on genetic and molecular characteristics to improve survival outcomes.
A gene therapy treatment has demonstrated significant promise in treating maple syrup urine disease (MSUD), a rare genetic disorder. The therapy prevented death and normalized growth in animal models, including a calf with the condition, and is being explored for potential use in humans.
Scientists discovered a protective variant of the HAQ-STING gene that prevents COPA Syndrome. This finding opens the door to a new gene therapy for the condition, which currently has no cure.
A novel bone marrow transplant process has been shown to be safe and curative for adults with sickle cell disease, offering a viable alternative to recent gene therapy products. The treatment, which uses a 'half-matched' donor, results in high cure rates and low side effects, making it a more accessible option for patients.
Researchers have discovered a novel gene therapy that can reverse conduction slowing and prevent cardiac arrhythmias by introducing the SCN10a-short gene into heart muscle cells. The treatment has shown promise in animal models and human cell studies, offering a potential solution for millions affected by arrhythmias worldwide.
A new genetic medicine has been developed to treat a rare genetic deficiency affecting the AIPL1 gene, causing severe retinal dystrophy. The treatment involves injecting healthy copies of the gene into the retina through keyhole surgery, resulting in dramatic improvements in sight for four young children.
Researchers at Virginia Tech have developed a method to convert gut bacteria into mini protein factories that produce and release sustained flows of targeted proteins within the lower intestine. This approach eliminates a major roadblock in delivering drugs to this part of the body, offering potential treatment for chronic diseases.
A new gene therapy has shown promise in treating a rare form of epilepsy, specifically Dravet syndrome, by replacing the SCN1B gene variant. The therapy increased survival rates, reduced seizure severity, and restored brain neuron excitability in mice with the condition.
The Rice University lab, in collaboration with Baylor College of Medicine, has developed a new gene-editing strategy called Repair Drive that improves the effectiveness of gene therapies in the liver. The technique enables the repair of liver cells at higher rates and equips them with a selective advantage to outcompete incorrectly edi...
Researchers at Virginia Tech have developed a way to convert gut bacteria into miniature protein factories that produce and release targeted proteins inside the lower intestine. This breakthrough could potentially treat chronic diseases.
Researchers discovered that mismatch repair genes are critical in eliciting damages to neurons vulnerable to Huntington's disease, triggering downstream pathologies and motor impairment. Targeting these genes may offer novel therapeutic approaches, including improving locomotor and gait deficits and reducing neuronal cell death.
Researchers at Osaka Metropolitan University assessed target genes in canine hepatocellular carcinoma (HCC) to develop molecular targeted therapies. The study identified potential gene targets, including PDGFB, which may improve treatment options for unresectable HCC.
Dr. Eric Levine's lab at UConn School of Medicine has been awarded $400,000 to study the genetic pathways of autism spectrum disorders using patient-derived human neurons. The team aims to identify genes and brain cell behavior that may cause these disorders, with potential applications for gene therapy.
Researchers developed a novel AAV-equipped nanomachine that successfully overcame gene therapy challenges in mice, including reduced efficiency due to neutralizing antibodies and hepatotoxicity. The nanomachine demonstrated sufficient gene transfer activity and suppressed liver toxicity markers.
Researchers have developed a new gene therapy that targets aggressive brain cancer, glioblastoma, with a precise delivery system. The treatment uses a novel virus to deliver a targeting drug to cancer cells, achieving cure rates of up to 90% in mouse models.
A new study adds weight to the safety and effectiveness of a gene therapy for hypophosphatasia, a rare inherited disorder that causes abnormal bone development. The treatment, AAV8-TNAP-D10, has shown promising results in mice models, with female mice achieving improvements in bone and teeth at lower doses.
Researchers have identified ALPK2 as a potential therapeutic target for treating heart failure with preserved ejection function (HFpEF). The enzyme is believed to prevent stiff heart conditions through regulating the TPM1 gene. This discovery offers new hope for developing treatment options targeting ALPK2.
The foundation has awarded eight recipients of the 2025 Damon Runyon-Rachleff Innovation Award, including five early-career researchers with initial grants of $400,000 over two years. The awardees aim to develop novel cancer therapies using innovative approaches such as engineered skin bacteria and small molecule-boosted drug delivery.
Batten disease, a neurodegenerative disorder, affects the bowel wall's enteric nervous system, causing degeneration and bowel symptoms. Gene therapy has been shown to slow symptoms and extend lifespan in mice by preventing enteric neuron degeneration.
Researchers at Johns Hopkins Medicine identified a new epigenetic approach to target colorectal cancer, using a mouse protein that disrupts cancer-causing chemical changes in genes. The study found that the protein, STELLA, can be used to develop a drug strategy to treat solid tumors.
Researchers developed a gene-editing treatment that reduces prion protein levels in the brain by up to 60% and extends mouse lifespan by about 50%. The base-editing approach could lead to a one-time treatment for prion disease patients.
Researchers investigated how two CAR T cells kill cancer with distinct signaling domains. CD28.ζ-CAR molecules work quickly and efficiently, while 4-1BB.ζ-CAR molecules linger in lipid rafts for sustained collaborative killing of tumor cells. This study aims to design CAR molecules maximizing antitumor activity beyond B cell malignancies.
Researchers have identified a 177-gene signature common to metastasis across cancers, allowing for personalized risk assessment and potential therapies. The discovery could lead to broader treatment options, faster drug access, and improved patient outcomes.
Researchers at UTHealth Houston have discovered two novel genes, DYRK1A and EGFR, linked to genetic mutations causing epileptic brain lesions. This breakthrough offers a new framework for understanding epilepsy and developing targeted therapies.
A zebrafish protein, Hmga1, has been found to unlock dormant genes for heart repair in mice. The discovery could lead to regenerative therapies to prevent heart failure in humans.
Researchers developed AI-driven therapeutic platform mimicking viral structures to deliver therapeutic genes to target cells. The innovative approach achieved precise symmetrical structures and effectively delivered payloads, paving the way for breakthroughs in gene therapies and next-generation vaccines.
Researchers have identified molecular changes that occur long before symptoms appear, shedding light on the development of Rett syndrome. These findings hold promise for developing safe gene therapies and monitoring biomarkers to track MECP2 gene function.
Researchers aim to advance eye transplantation through cutting-edge technologies and techniques, including cell-based therapies and device development. The six-year project, led by Kia Washington at the University of Colorado Anschutz Medical Campus, seeks to overcome current limitations in whole-eye transplant success rates.
A new study shows targeted delivery of energy-disrupting gene therapy using nanoparticles shrinks glioblastoma brain tumors and aggressive breast cancer tumors in mice. The technology, mLumiOpto, induces light-activated electrical currents inside cells to disrupt mitochondria, leading to programmed cell death and DNA damage.
Researchers have developed a new generation of cell-penetrating antibodies that can target cancer cells and deliver therapeutic molecules directly into tumor cells. The 3E10 antibody shows great promise for treating cancers with defective DNA repair pathways.
A new gene therapy has reversed the effects of heart failure in a large animal model by increasing blood pumping efficiency and dramatically improving survival rates. The therapy restored critical functions of heart cells and improved heart function on the microscopic level.
Researchers have identified new potential therapeutic targets for diabetic kidney disease (DKD), a leading cause of kidney failure. The study reveals common and cell-type-specific changes caused by insulin-resistance, representing new targets for pharmacological or targeted gene therapy approaches.
Branden Baptiste, 20, becomes the world's first patient to receive base editing gene therapy for sickle cell disease. The treatment has left him feeling 'more than fine' with no symptoms of the disease. After undergoing chemotherapy and a series of tests, Branden was infused with genetically treated cells in December 2023, allowing his...
A new gene therapy has shown promise in reversing a condition that causes significant stillbirths and premature deliveries globally. The therapy, developed by University of Florida Health researcher Helen N. Jones, successfully boosted placental function and delivered normal-weight offspring in guinea pig models.
A Phase 2 clinical trial has been initiated by Genethon and Hansa Biopharma to evaluate the efficacy and safety of imlifidase, a gene therapy for Crigler-Najjar syndrome. The trial aims to address anti-AAV antibodies, which prevent up to 1 in 3 people from benefiting from gene therapies.
Researchers at UCSF are enrolling patients in a clinical trial to correct the genetic mutation causing sickle cell disease using non-viral CRISPR-Cas9 gene editing. The therapy aims to eliminate the need for a bone marrow transplant and create a new blood system free of the disease.
Positive initial results from Genethon's gene therapy GNT0004 show stabilization of motor functions and improved dystrophin expression in patients with Duchenne Muscular Dystrophy. The therapy is expected to be launched in pivotal trial phases in Europe and the US in Q2/2025.
A new RNA-based technology called StitchR facilitates effective use of gene therapy for difficult-to-treat, large-gene diseases like muscular dystrophies. It delivers two halves of a gene separately, resulting in seamless reconstitution of large mRNA in affected tissues.
Researchers discovered a protective inherited mutation in the ADNP gene that enhances protein interactions and offers protection against developmental disorders. The study challenges previous assumptions about genetic mutations and their impact on brain development.
More than 70 hematology researchers from the University of Miami Miller School of Medicine will showcase their work at the 66th ASH Annual Meeting & Exposition. Researchers from Sylvester Comprehensive Cancer Center are authors or co-authors on a significant number of posters presented during the event.
Researchers from UAB have developed a gene therapy that reverses metabolic dysfunction-associated steatohepatitis (MASH) in mice, and most obese and type 2 diabetic patients could benefit from this treatment. The therapy, based on the fibroblast growth factor 21 protein, mediates long-term reversal of liver fibrosis and MASH.
The institute's Spark Grant program recognizes innovative research in gene and cell therapy, with recipients working on projects spanning disease fields such as neuroinflammation, cancer, and tuberous sclerosis. The $1.15M grant will support the development of novel treatments and commercialization outcomes.
Researchers have developed a groundbreaking gene therapy that targets Diamond-Blackfan anemia, a rare genetic blood disorder. The treatment uses regulated GATA1 expression to correct multiple genetic mutations with a single vector, offering new hope for patients currently reliant on lifelong follow-up care and stem cell transplants.
The CRISPR tool was successfully used to correct a genetic defect in cells affected by chronic granulomatous disease. However, the repair process also introduced new genetic defects, highlighting the need for caution when using CRISPR technology in clinical settings.
The US Department of Defense has awarded $514,000 to study the efficacy of gene editors in treating Duchenne Muscular Dystrophy. Researchers will explore non-viral options for delivering gene therapy through 'self-delivering' gene editors, aiming to improve safety and efficacy.
A new study in mice shows a unique mRNA delivery method can successfully edit faulty genes in fetal brain cells. The technology has the potential to stop progression of genetic-based neurodevelopmental conditions like Angelman syndrome and Rett syndrome before birth.
The ASHG 2024 Annual Meeting will showcase the latest research in human genetics and genomics. The event will feature a Presidential Symposium on Mendelian traits and a Distinguished Speakers Symposium on the promise of human genetics and genomics, among other sessions.
Researchers aim to improve gene therapy design for life-long correction of genetic diseases, but face unknowns including immune response and genomic changes. A five-year NIH award will fund an analysis of genetic and cellular determinants of gene therapy longevity.
Researchers have identified a protein called PERM1 that regulates both energy and heart muscle contraction, offering a new therapeutic approach to systolic heart failure. By addressing the underlying problem of weakened heart muscle, PERM1 may help restore cardiac function and improve patient outcomes.
A large-scale study of children with genetic disorders found that thousands benefited from targeted treatments and support after receiving a genetic diagnosis, with over 20% able to start or adjust therapies. Researchers expect this number to grow as new genetic therapies develop.
A clinical trial of 32 patients with cerebral adrenoleukodystrophy found that six years after treatment, most remained free of major disabilities. However, the study also highlighted safety concerns about blood cancers post-treatment, including myelodysplastic syndrome and acute myeloid leukemia.
Researchers have developed a novel gene therapy approach that targets and breaks down faulty ribonucleic acids in the KCNA2 gene, which is associated with recurring seizures. The therapy has shown promise in reducing excessive neuron activity linked to epilepsy.
Researchers review UBA1 loss of function in VEXAS Syndrome, a hematoinflammatory disorder characterized by severe inflammation, cytopenias, and oncogenicity. They explore therapeutic options, including clone-targeting drugs, to combat this challenging disease.
Two UCF cancer researchers, Alicja Copik and Debbie Altomare, have received $100,000 grants from the Florida Breast Cancer Foundation to develop new treatments for breast cancer. They focus on enhancing natural killer cells to fight cancer and harnessing the body's immune system to create new therapies.
Researchers developed a novel approach to optimizing siRNA-loaded lipid nanoparticles using NMR-based molecular-level characterization. Pre-mixed LNPs exhibit superior gene-silencing effects due to a stacked bilayer structure that enhances gene silencing.
A single-dose gene therapy has been shown to significantly reduce bleeding episodes in adults with hemophilia B, with an average reduction of 71% compared to standard treatment. The therapy, which enables the liver to produce clotting factor IX, has been FDA-approved for use in patients with this genetic disorder.
Researchers discovered that DNA methylation patterns, like cellular memory markers, prevent reprogrammed cells from fully adopting new identities. This limitation limits the effectiveness of long-term treatments and therapies.
A new NIH-funded project aims to address gene therapy's ethical and policy challenges, gathering information from experts in six domains. The study will provide recommendations for addressing these challenges, with implications for patient safety and health outcomes.
Scientists have developed new therapies that selectively remove aggregated tau proteins associated with Alzheimer's disease in mice. The approach utilises TRIM21 to target tau aggregates, leaving healthy tau intact, and demonstrates potential for other brain disorders driven by protein aggregation.