Articles tagged with Gene Therapy
A team of UPenn researchers is developing a new CAR T-cell gene therapy treatment for advanced metastatic prostate cancer, with the goal of overcoming resistance to current treatments. The ACGT-funded study aims to re-engineer T-cells to induce safe, long-term remission in patients.
Researchers analyzed ovarian cells from young and old non-human primates to understand ovarian aging. They identified genes that could be used as biomarkers and point to therapeutic targets for diagnosing and treating female infertility and age-associated ovarian diseases.
Researchers identified specific regions of chromosome 21 causing memory and decision-making problems in mice with Down syndrome. The study found that different gene groups on mouse chromosomes 16, 10, and 17 contribute to cognitive issues.
Researchers at UCLA and international partners have successfully treated nine patients with X-linked chronic granulomatous disease (X-CGD) using a stem cell gene therapy. Six patients are now in remission and have stopped other treatments, with no complications reported.
A gene therapy trial conducted in US and UK patients with X-linked Chronic Septic Granulomatosis, a rare genetic disease causing severe infections, has shown success. Six of nine patients are free of treatment complications, offering hope for sustainable treatment.
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.
A recent study published in PNAS reveals that extracellular matrix protein-1 (ECM1) plays a crucial role in the pathogenesis of ulcerative colitis, a type of inflammatory bowel disease. The researchers found that ECM1 is highly expressed in macrophages and regulates their polarization through the GM-CSF/STAT5 signaling pathway.
A study found that an overactive immune system gene causes schizophrenia-like changes in mice, including neural and behavioral aspects of the disease. The researchers suggest that this gene may be contributing to the development of schizophrenia through mis-regulated synaptic pruning.
Researchers have identified a second gene, BSND, which determines the severity of kidney disease in patients with Joubert syndrome. The discovery has significant implications for diagnosis and treatment of genetic kidney diseases, offering hope for personalized therapies to reduce disease severity.
A study found that APLN overexpression correlates with worsened prognosis in ovarian cancer patients treated with bevacizumab. The researchers also identified a distinct gene signature associated with resistance development, paving the way for new treatment strategies.
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 Michigan Technological University have developed a single-particle method using atomic force microscopy to determine virus isoelectric points, improving the accuracy of characterization and paving the way for more efficient vaccine production and gene therapy manufacturing.
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.
A breakthrough gene therapy treatment has been shown to offer long-term benefits for people with Haemophilia A, transforming the lives of 13 UK patients. The treatment resulted in a substantial fall in bleeding rates three years after treatment, with none requiring regular factor VIII injections.
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 have identified a new subtype of satellite cells that can regenerate muscle tissue without the transcription factor PAX7. This discovery could lead to new gene therapies for people with muscular dystrophy, such as Lavin, who has a rare genetic mutation preventing her from producing this protein.
Scientists have developed a new gene-therapy technique using exosomes to communicate with other cells, which has shown promising results in slowing tumor growth and prolonging survival in mice with gliomas. The technology relies on patented technology that prompts human cells to produce nanocarriers containing a drug.
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 recent study reveals a new link between the pathways involved in RNA degradation, identifying a critical protein called RPT2a that targets RNA quality control proteins for degradation. This finding has potential applications in antiviral development, gene therapy, and agriculture.
A mutated gene, USP9X, regulates a network of genes underlying Intellectual Disability and Autism Spectrum Disorder. Focusing on this network may lead to therapy developments for neurological disorders.
Researchers at Wyss Institute for Biologically Inspired Engineering developed an AAV-based gene therapy that targets leaky lung cells by overexpressing the CD98hc protein. This approach shows great promise in reducing vascular leakage and improving lung function, offering a new hope for treating pulmonary edema.
Researchers at Harvard's Wyss Institute have developed a high-throughput synthetic biology approach to improve AAV capsid proteins, revealing hidden features and potential new accessory proteins that could help fast-track future gene therapies. The study uses machine-guided design to generate large numbers of high-quality capsid variants.
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.
A new gene therapy aims to 'switch off' genes that promote cardiovascular disease by reducing fatty material buildup in arteries. The treatment, developed at the University of Sheffield, has the potential to slow or stop the progression of heart disease.
Researchers identified genetic factors and parasite numbers that predict treatment success in cutaneous leishmaniasis. A small difference in parasite numbers makes a big difference in patient response.
Researchers at Martin-Luther-Universität Halle-Wittenberg have developed new DNA transporters using artificial fats, offering a promising alternative to modified viruses. The lipids can encapsulate nucleic acids, protect them from degradation, and introduce them into cells efficiently.
Scientists have characterised genetic alterations caused by six cancer therapies, revealing 'mutational footprints' that can help optimize treatment efficacy and minimize side effects. The study provides a new understanding of the relationship between therapy-induced mutations and long-term side effects.
Researchers from Boston University School of Medicine have developed a novel computational method, iEDGE, which identified several candidate breast cancer drivers and multiple pan-cancer drivers, including TRIP13 and TPX2.
A new study shows that a commonly used vector for large gene transfer can deliver genes to retinal cells, but provokes an acute inflammatory response when injected subretinally into rats. The researchers conclude that further work is needed to understand the inflammatory pathways involved and modulate the immune response.
A recent study by Goethe University Frankfurt identified 17 microRNA families that play a crucial role in the interface between the immune and neuronal systems, showing sex-specific differences in gene expression. These findings may lead to new therapeutic approaches for psychiatric diseases.
Patients with diverse HLA genes respond better to immune checkpoint inhibitors, surviving longer with improved treatment outcomes.
Researchers at Penn State College of Medicine developed a new method to model gene interactions and predict changes over time. The idopNetwork can create personalized networks for individual patients, showing complex gene connections and predicting outcomes.
Researchers developed new imaging methods to study retinal cilia, discovering previously unknown compartments and pinpointing protein locations in diseased cilia. These findings contribute to a better understanding of structural defects leading to cilia-related blindness.
Researchers developed a gene therapy combining FGF21 and ?Klotho treatments to target multiple age-related diseases. The single-formulation treatment successfully treated obesity, type II diabetes, heart failure, and renal failure in mice.
Researchers developed a combination gene therapy treating four age-related diseases in mice, including heart failure, kidney failure, diabetes, and obesity. The treatment showed significant improvements, reversing or completely eliminating disease symptoms in obese and diabetic mice.
A three-drug combination improved lung function and reduced symptoms in patients with the most common genetic mutation for cystic fibrosis. The therapy, approved by the FDA, targets the underlying cause of the disease and demonstrated significant benefits in lung function and quality of life.
A comprehensive genome sequencing study reveals rare CFTR mutations in Puerto Rican and Dominican patients, driving the disease's progression. This finding underscores the need for diverse clinical trials to develop personalized therapies, as transformative new drugs may not benefit minority populations.
A new gene therapy has shown promise in suppressing epileptic seizures in animal models and human tissue samples. The treatment delivers a specific gene that produces dynorphin, modulating neural activity and dampening seizure spread.
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.
Researchers have discovered a genetic mutation in monkeys that causes Bardet-Biedl Syndrome, a devastating eye disorder. The discovery provides a promising animal model for developing gene and cell therapies to treat the condition in humans.
Dr. Jennifer J. Lentz and her team will research a treatment for vision loss in Usher using antisense oligonucleotides, targeting the human 216A mutation in the USH1C gene. The goal is to develop an effective treatment for vision loss in Usher Syndrome.
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.
A plant-based compound closely related to resveratrol has been found to enhance gene delivery to hematopoietic stem cells, reducing antiviral restriction at endosomes. This breakthrough enables faster and more efficient treatments for diseases such as sickle cell anemia and leukemia.
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
Researchers used fruit flies to study the genetic mechanisms behind ADHD and identified areas of the genome that influence response to treatment. This discovery has significant implications for the development of personalized medicine, enabling tailored treatments based on individual genetic profiles.
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.
Researchers have developed a new approach to delivering sight-saving gene therapy to the retina, using a small needle to inject harmless viruses into the suprachoroidal space. This method could provide a more permanent therapeutic option for patients with common diseases such as wet age-related macular degeneration and inherited retina...
Researchers identified genetic variants related to Parkinson's disease symptom onset, which could enable delimiting research on new therapeutic targets. The study, led by Cristina Malagelada, used biocomputing techniques and analyzed statistical interactions to identify genetic associations.
Researchers from King's College London and UCL have identified 44 genes linked to age-related hearing loss, offering a clearer understanding of the condition's development. The study's findings may lead to new therapies for millions worldwide affected by hearing loss as they age.
Researchers at George Washington University found a potential therapeutic target for prostate cancers with PTEN mutation by identifying the connection between PTEN and ARID4B genes. Suppressing ARID4B expression in cancer cells with PTEN mutation significantly inhibits cell growth and increases cell death.
Researchers at Neuroscience 2019 announce new gene therapy research promising to prevent, treat, and reverse incurable neurological disorders. Successes in restoring lost functions in animal models of neurological diseases are discussed, as well as technique advancements that may improve future treatments.
Researchers at RMIT University have developed a non-viral gene delivery method that has proven effective in laboratory tests, opening the door to treatment of various genetic diseases. The method uses Metal Organic Frameworks (MOFs) and could significantly reduce the time and expense involved in bringing new treatments to market.
A novel gene therapy treatment has shown promising results in patients with glycogen storage disease, allowing them to discontinue their reliance on cornstarch and experience normal blood glucose levels. Patients have reported significant improvements in energy, muscle strength, and overall quality of life.
Boston University School of Medicine researchers have developed a new protocol to produce large quantities of AAVs, which can deliver specific genes into humans and animals. The advanced technique bypasses developmental effects, saving time and reducing the number of animals used in research.
After a tragic death in 1999, gene therapy faced a setback, but James Wilson's rediscovery of adeno-associated viruses (AAVs) paved the way for its resurgence. Today, AAVs are used in nearly 100 drug development programs and have been approved by the FDA for treating a fatal neurological disease.