Articles tagged with Gene Therapy
A clinical trial using genetic testing to guide antiplatelet medication found a 34% reduction in serious cardiovascular events at one year, as well as a significant decrease in the number of events per patient. This finding suggests that genetically guided therapy may be beneficial for patients with specific genetic variants.
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.
Using high-throughput screening of adeno-associated viral (AAV) vector capsid libraries, researchers identified functional and efficient AAV variants after only one round of selection. Infection with a high multiplicity of infection (MOI) was found to be preferable to infection with a low MOI, reducing variation between screens.
Researchers at Boston Children's Hospital have successfully used gene therapy to reverse cardiac dysfunction and scarring in two mouse models of Barth syndrome. The treatment, which replaced the mutated TAZ gene, prevented progressive cardiomyopathy and restored heart function in both whole-body and heart-only TAZ deletion mice.
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.
Scientists from the University of Illinois at Urbana-Champaign developed a new CRISPR gene-editing methodology that inactivated a key gene responsible for ALS, slowing disease progression and improving muscle function. The treatment also increased overall survival in mice with aggressive forms of ALS.
Duchenne muscular dystrophy researchers have identified a potential new method for therapies, using small molecules to increase utrophin protein production. This approach bypasses the immune system's challenges and may provide functional characteristics similar to dystrophin.
The Clinic for Special Children's SMA Prevention Readiness program successfully identified 318 carriers and 9 affected couples, treating 3 infants with gene therapy. The program's cascade testing approach was effective in detecting genetic risk, leveraging funding from biotech company AveXis.
Researchers at Karolinska Institutet have discovered a gene, Synaptotagmin 13, that protects against motor neuron degeneration in ALS and SMA. Gene therapy was shown to increase life expectancy in animal models.
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 clinical research program at Columbia University Irving Medical Center will treat eight more ALS patients with FUS mutations using the investigational therapy jacifusen. The program aims to expand access to treatments for rare ALS subtypes associated with gene mutations.
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.
Scientists have developed a simple and precise cancer therapy approach based on selective electrochemical generation of H2 in tumor regions. The H2-ECT method enables large-scale tumor therapy with ideal selectivity and targeting to kill tumors with minimal damage to normal tissue.
Researchers at St. Jude Children's Research Hospital are exploring genetically engineered immune cells to attack pediatric sarcoma using a new ACGT grant. The approach aims to target not only cancer cells but also blood vessels supporting tumor growth, offering a promising new treatment for children with solid tumors.
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 the University of Missouri have made a groundbreaking discovery by identifying the genetic mutation responsible for Chédiak-Higashi syndrome in domestic cats. This finding has significant implications for treating inherited diseases and preventing their transmission to future generations.
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 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 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 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.
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.
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 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.
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 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.
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 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.
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.
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.