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.
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.
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.
Scientists at ChristianaCare's Gene Editing Institute are developing a novel gene therapy for inherited blood disorders like sickle cell disease using CRISPR technology. The team aims to identify genetic variations that affect treatment efficacy and create a tailored approach for personalized medicine.
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.
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 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 inducing liver regeneration with thyroid hormone boosted CRISPR/Cas9-mediated gene correction, achieving 10.8% and 3.5% efficiency rates in neonatal and adult mice respectively. This discovery could lead to more efficient gene therapy for human diseases.
A new study from Penn Medicine found that proton therapy reduces the risk of radiation-induced heart diseases in lung cancer patients. Mini-strokes and heart attacks were significantly less common among patients who underwent proton therapy compared to those treated with conventional photon-based radiation therapy.
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 UNC School of Medicine show that gene editing with CRISPR-Cas9 can restore function in an animal model of Angelman syndrome. The therapy was effective in restoring the UBE3A enzyme in human neurons and treating deficits in an animal model, offering a long-lasting treatment or cure for this debilitating disease.
A genotype-agnostic gene therapy for cystic fibrosis has shown promise in clinical trials, potentially treating the disease in any patient, independent of their underlying mutation. Challenges remain to be overcome, including developing effective drug delivery systems that can reach pulmonary epithelial cells at low doses.
A Yale professor has received a $500,000 grant to develop a versatile and highly scalable strategy for treating pancreatic cancer. The technology, called MAEGI, targets multiple differences in cancer cells and activates multiple immune system responses.
Researchers at UMD discovered that mosquitoes lack a critical gene for proper body segmentation, but a related gene took its place. This finding highlights the importance of caution in genetic studies and offers new potential avenues for targeted mosquito control strategies.
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.
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
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 at Keck School of Medicine of USC have received a five-year, $14.6 million grant to advance gene therapy for HIV control without daily medicines. The approach is inspired by three cases of HIV cure and aims to prepare patients for stem cell transplants with little toxicity.
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...
A McGill-led study reveals that suppressing the OSMR gene can improve radiation therapy effectiveness and expand lifespan in preclinical mouse models. Glioblastoma's resistance to therapy is overcome by starving cancer stem cells with energy production halted.
Researchers developed novel variants of adeno-associated viral (AAV) capsids with improved transduction properties in the mouse retina and cornea. The efficient gene delivery of these variants was confirmed in non-human primate tissue, adding to their potential use in treating human ocular diseases.
A breakthrough study using gene therapy to target the inner retina has prevented blindness in a mouse model of CLN3 Batten disease. The treatment led to significant survival of bipolar cells and preserved retinal function, according to researchers.
Researchers successfully deliver gene therapy using nanoparticles to inhibit abnormal blood vessel growth in rats and mice eyes, providing evidence for treating wet age-related macular degeneration and inherited retinal diseases.
A recent report of two children's deaths in a gene therapy trial for X-linked myotubular myopathy has sparked concerns over the safety of gene therapy vectors. The editorial emphasizes the need for iterative development and cooperation among scientists to ensure safe and effective treatments for rare genetic diseases.
Researchers have developed a reagent for selective and safe coating of the liver sinusoidal walls to control clearance of gene therapy drugs. The coating agent improved gene transfer efficacy by 2-4 times to the myocardium and skeletal muscles, and 10 times to colorectal cancer, reducing medical costs and adverse effects.
A study published in JAMA Neurology reports successful delivery of micro-dystrophin to patients with Duchenne muscular dystrophy (DMD), resulting in functional improvements. The therapy showed robust gene expression and localization, as well as improved muscle function measured by North Star Ambulatory Assessment scores.
Researchers found that ovarian cancer patients with a modified BRCA1 gene do not respond better to platinum chemotherapy or have a better prognosis than those with the normal functioning gene. However, they did live longer on these treatments compared to those without any gene modifications.
A research team from Genethon has successfully inhibited the immune response induced by AAV antibodies, paving the way for repeated administration of gene therapy treatments. This breakthrough could enable treatment of rare genetic diseases and improve patient outcomes.
Scientists at ChristianaCare's Gene Editing Institute have developed a new CRISPR advance that can safely target and disable the NRF2 gene linked to a bleak prognosis in lung cancer tumors. This approach aims to improve the efficacy of conventional chemotherapy and radiation treatments while minimizing harm to normal cells.
Research identified potentially treatable genetic mutations in prostate cancer patients who did not respond to PSMA-targeted therapy. Targeted next-generation gene sequencing found mutations in six out of seven patients, including alterations in DNA damage-repair genes such as TP53, ATM, and CHEK2.
A study in mice suggests gene therapy can build significant muscle mass quickly and reduce the severity of osteoarthritis, even without exercise. The therapy also prevented obesity and improved cardiovascular health.
The National Gene Vector Biorepository (NGVB) provides valuable resources to gene therapy investigators, including 93 unique reagents and a searchable database of animal safety studies. These resources aim to decrease compliance risks, address clinical trial funding periods, and reduce costs.
Researchers at University of California San Diego School of Medicine identified a novel way to treat Danon disease using gene therapy, adding a specially designed gene that restores LAMP2 function. The treatment improved cardiac and liver function in mice with the disease, offering a new approach beyond heart transplants.
A systematic review and meta-analysis found that dual therapy with a direct oral anticoagulant (DOAC) plus P2Y12 inhibitor was associated with reduced risk of major bleeding compared to triple therapy with a vitamin K antagonist (VKA) plus aspirin and P2Y12 inhibitor for patients with nonvalvular atrial fibrillation after percutaneous ...
OCU400, a novel gene therapy product candidate, demonstrates efficacy in rescuing photoreceptors from degeneration by resetting retinal homeostasis. The treatment has the potential to be broadly effective across genetically diverse IRDs, offering hope for millions of people worldwide affected by inherited retinal degenerations.
A novel gene therapy has been developed to regenerate functional new neurons in mouse models of Huntington's Disease, offering a potential treatment for the condition. The therapy uses NeuroD1-based gene therapies to convert brain internal glial cells into functional new neurons.
Researchers delivered an ALDH2 gene to mice with the deficiency, using a virus-mediated approach. The treated mice showed no signs of the acute abnormalities or chronic disorders normally associated with ethanol exposure in ALDH2 deficiency.
A team of scientists from Purdue University and international research institutions developed a new method for identifying nonviral vectors in gene therapies. This approach uses big data, patent, and clinical data mining to uncover emerging trends in the field. The study aims to guide future developments in gene therapy.
Researchers have developed a new single AAV gene therapy platform that can treat almost any mutation, showing improved vision in blind mice and paving the way for clinical trials by 2025.
Researchers at Technical University of Munich used CRISPR-Cas9 gene scissors to correct the mutated dystrophin gene in living pigs, improving muscle function and life expectancy. The therapy has shown promising results in a clinically relevant large animal model, mirroring Duchenne muscular dystrophy in humans.
A team at Massachusetts Eye and Ear has identified GPR108, a G protein-coupled receptor, as a molecular 'lock' necessary for most adeno-associated virus (AAV) vectors to gain access to cells. This discovery may enable scientists to better explain, predict, and ultimately direct AAV gene transfers to specific tissues.
Researchers develop biodegradable nanoparticles that target and kill pediatric brain tumor cells in mice, surviving 20-63% longer than untreated mice. The treatment uses a combination of the suicide gene and ganciclovir, showing promise for new therapies targeting these deadly brain malignancies.
Researchers at the Mayo Clinic have developed a single-dose gene therapy to treat cocaine addiction. The therapy uses a recombinant adeno-associated viral vector to deliver a gene that metabolizes cocaine into harmless byproducts.
Researchers developed an adeno-associated virus vector to deliver anti-pTau antibodies directly into the hippocampus of mouse models with CTE, reducing pTau levels across the CNS. The study suggests this strategy could be effective in humans and may offer a new treatment option for CTE.
Researchers at Mayo Clinic tested a new injection method for delivering gene therapy vectors directly into the kidney, outperforming traditional intravenous injections. The study found that direct injections were superior to intravenous injections and had fewer off-target effects.
Scientists at Scripps Research Institute have developed a molecular switch that enables precise control of gene therapy doses. The technique involves embedding an RNA molecule called a hammerhead ribozyme into the genes used in gene therapies, allowing doctors to regulate the dosing level.
Researchers at the University of Illinois have developed a new technique to increase CRISPR-Cas9 efficiency, achieving up to five times higher rates of inserting genes into human cells. This breakthrough has significant implications for clinical gene-therapy applications and basic biological research.
Neuroscientists at Lund University have developed a new technology to deliver gene therapy to specific cell types, accelerating evolution from millions of years to weeks. The method combines computer simulations and modeling with gene technology and sequencing to tailor virus shells for precise targeting.
A team of scientists has developed an artificial intelligence approach to engineer improved AAV capsids for gene therapy delivery. The research reveals the existence of a previously unknown protein and demonstrates the potential to transform gene therapy. The study's findings have significant implications for the future of gene therapy.
Gene therapy approaches are being developed to treat a variety of inherited neurometabolic diseases, including X-linked adrenoleukodystrophy and mucopolysaccharidoses. Microglia are emerging key players in these diseases and are targeted for therapeutic efficacy.
A recent study published in JAMA Network Open reveals that analyzing gene expression in tumor cells can reveal new targets for therapy in pediatric cancer patients. The study found that 68% of children with cancer showed potential for treatment through gene expression analysis, compared to 46% who responded to DNA mutation analysis.
Stephan Grupp, a leading pediatric oncologist, has been elected to the National Academy of Medicine for his groundbreaking work in cell and gene therapy. He is recognized for developing an entirely novel therapy for acute lymphoblastic leukemia and leading the first global engineered cell therapy trial.
Researchers found gene therapy potential in treating neurological disorders like Alzheimer's disease, amyotrophic lateral sclerosis, and Parkinson's disease. New therapeutic approaches reprogram glial cells into motor neurons and deliver antibodies to protect against toxins.
Researchers have created a mouse model with a fully replaced human version of the MAPT gene, allowing for accurate genetic therapy development and evaluation. This breakthrough enables scientists to study the role of the human version of the gene in humans and develop therapies.
Researchers have made significant progress in developing gene therapy to treat wet age-related macular degeneration (AMD). Six patients have maintained vision after receiving a single intravitreal injection of gene therapy, offering hope for a potential
A Penn Medicine study shows delivering a 'distant cousin' of a key protein prevents muscle damage and maintains muscle function in both small and large animal models. The synthetic substitute, utrophin, proved to be an effective and safe alternative to dystrophin, with significant implications for gene therapy.