Research on treating disease by editing or delivering genes. Covers sickle-cell gene therapy, gene-editing nanoparticles, mRNA delivery, and blood-cell-inspired carriers.
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.
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 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.
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.
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.
Researchers have identified acid ceramidase as a key enzyme in the production of psychosine, a toxic glycolipid that accumulates in people with Krabbe disease. Removing or inhibiting this enzyme has been shown to decrease or eliminate psychosine production and disease symptoms.
Researchers at Michigan State University have developed a new approach to delivering enzyme-producing genes that can convert certain drugs into toxic agents and target tumors. The study found that nano-bubbles, or extracellular vesicles, were 14 times more effective at delivery and killed over half of the breast cancer cells in mice.
The team aims to understand how genetics contributes to driving aggressive pathology in prostate cancer by combining pathology, computational modeling, and genomics. They plan to identify therapeutic options by testing combinations of genes in mouse models of the disease.
A Nemours study suggests genetic testing can personalize PPI dosing for eosinophilic esophagitis patients. This could improve efficacy and reduce side effects in up to 90% of children, according to researchers.
Researchers have developed a new gene therapy that converts glial cells into neurons, improving motor function in mice and potentially treating stroke. The treatment uses the NeuroD1 gene and has been shown to increase neuronal density and reduce brain tissue loss in mouse models of stroke.
Researchers used a canine model of Leber congenital amaurosis to study the effects of gene therapy on photoreceptor cells. They found that dogs treated before 63% of their cells were lost had lasting improvements, while those treated later experienced continued degeneration.
Administering nanoparticles carrying messenger RNA for the arginase gene restored urea cycle function and prolonged lifespan in genetically deficient mice. This treatment approach holds promise for treating inherited metabolic disorders like arginase deficiency.
Researchers at Ohio State University have made significant breakthroughs in gene replacement therapy for Niemann-Pick type A disease, demonstrating its safety and therapeutic effects in nonhuman primates and mouse models. The treatment has shown a 'bystander effect', preventing motor and memory impairment and increasing survival rates.
Researchers at Cardiff University have identified the CYFIP1 gene as a potential cause of abnormalities in brain structure seen in psychiatric conditions. The study found that the deletion of this gene leads to thinning of the insulation around nerve cells, disrupting communication between brain regions.
A clinical trial found that an oral activator, veledimex, controlled the transcription of human interleukin-12, reducing toxicity while preserving anti-tumor effects. Patients with recurrent glioblastoma had improved overall survival rates, with some experiencing up to 17.8 months of survival.
Researchers used an optimized AAV9 vector to deliver the galactosylceramidase gene to a mouse model of Krabbe disease, improving clinical symptoms and prolonging median survival. The treated mice lived up to 150 days, compared to 41 days for untreated mice.
A new study identifies an enzyme critical for normal wound healing in diabetes, suggesting a potential treatment with allopurinol. In diabetic mice, blocking uric acid production improved healing rates.
Huda Zoghbi, MD, is recognized for her contributions to the field of human genetics, including discoveries of genes responsible for Rett syndrome, spinocerebellar ataxia type 1, and other conditions. Her work has enriched the development of human genetics and its applications in science, medicine, and health.
Scientists have discovered that microRNAs play a crucial role in regulating genes in cell nuclei, not just cytoplasm. MicroRNAs targeting the nucleus can increase gene expression, offering a novel approach to gene therapy.
Scientists at Boston Children's Hospital create human tissue models of CPVT and reverse the condition using gene therapy in a mouse model. The study opens up possibilities for single-dose gene therapy treatments for inherited arrhythmias like atrial fibrillation.
Researchers have identified a gene variant associated with longevity and found it can prevent atherosclerosis by rejuvenating blood vessels. Studies on animal models and human patients showed improved endothelial function and reduced inflammation.
Researchers found that the retina can rebuild itself after treatment, recovering normal light responses and connections to other neurons. Gene therapy successfully restored rod photoreceptors' function in a mouse model with genetically defective rods.