Articles tagged with Gene Therapy
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.
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.
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.
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.
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.
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 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.
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.
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 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.
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.
A defective degradation enzyme leads to the accumulation of gangliosides, causing deterioration in patients with storage diseases. Researchers have discovered that genetic disorders can trigger a cascade of consequential damages.
Researchers have successfully developed and tested a gene therapy approach using CRISPR-Cas9 technology to treat Steinert's myotonic dystrophy, a devastating neuromuscular disease. The study showed that the expanded CTG triplet repeat in the DMPK gene was 'cut' and removed from the gene, reducing toxic RNA aggregates in muscle cells.
A recent study by the University of Granada's Tissue Engineering Research Group has found that resident hospital doctors specialising in Family Medicine lack sufficient training on advanced therapies. The researchers also discovered a positive attitude among these doctors towards the use and application of such therapies.
A new roadmap by the Engineering Biology Research Consortium outlines potential breakthroughs in synthetic biology, including genetically modified crops, disease-fighting microbes, and novel biofuels. The report aims to secure federal support for this field to address societal challenges and fuel economic growth.
A team of researchers delivered a therapeutic gene to the spinal canal of infant rhesus monkeys, resulting in sustained expression of the alpha-I-iduronidase enzyme. The study's findings suggest a promising approach for treating severe forms of neuropathic storage diseases.
Scientists at ETH Zurich found that a cellular mechanism called SCF detects and targets alpha-synuclein fibrils for breakdown. This mechanism could be used to develop therapies for neurodegenerative diseases like Parkinson's. Gene therapy and stem cell transplantation may also offer new options.
Researchers have identified a potential therapy for limb girdle muscular dystrophy using lithium to improve muscle size and strength in mice. The findings suggest that inhibiting the protein GSK3beta with lithium chloride can lead to significant improvements in mouse strength and muscle mass.
A new statistical machine learning framework has been developed to reconstruct the original network after a viral attack, which is crucial for designing new medicines or gene therapies against viruses and diseases like cancer. The framework actively incorporates the influence and causality of the attack into its learning algorithm.
Scientists from Sanford Burnham Prebys have revealed a new mechanism for ALS' pathogenesis and suggest that modulating membralin has potential in ALS therapy. A membralin-boosting gene therapy extended the survival of mice with ALS-like symptoms, providing an important new perspective into the disease.
The Children's Tumor Foundation has launched the NF2 Accelerator Initiative, a three-year research effort to bring active NF2 treatments to patients. The initiative focuses on expanding the clinical drug pipeline, improving drug selection through innovative testing models, and developing gene therapy options.
A new clinical trial at IU School of Medicine shows that Burosumab significantly alleviates symptoms of X-linked hypophosphatemia, a phosphate-wasting disease causing rickets and osteomalacia. The study found substantial healing in 72% of children treated with Burosumab versus 6% in the conventional therapy group.
Scientists at CU Anschutz Medical Campus identified a rare genetic mutation causing an immune dysregulation syndrome, characterized by recurrent infections and autoimmune complications. The discovery provides unique insights into cell biology and suggests the possibility of treating the disease with gene therapy.
A study of 429 men with metastatic castration-resistant prostate cancer found correlations between specific genetic alterations and clinical outcomes. The loss of the RB1 tumor suppressor gene was significantly correlated with poor survival in patients treated with androgen receptor signaling inhibitors.
Researchers have developed an algorithm that can predict synthetic lethal interactions between genes, which could lead to more targeted and effective cancer treatments. The Slant algorithm uses data from existing protein networks to identify patterns associated with these interactions.
A new experimental therapy has demonstrated potential in slowing the progression of a genetic form of ALS by reducing SOD1 protein levels. Patients who received the treatment showed improved breathing capacity, muscle strength, and functional abilities compared to those given a placebo.
A researcher at Oregon State University has received a five-year, $3.3 million grant to develop a way for cystic fibrosis patients to get molecular treatment via an inhaler. The treatment would work on every patient, regardless of their genetic mutation, and could vastly improve existing therapeutic methods.
Scientists discovered that sex and diet substantially affect the proteome, a collection of proteins in an animal cell. Understanding these interactions may lead to personalized treatments for humans. The study analyzed large public datasets on human and mouse proteotypes, diet, and genetic status.
A new study compares the effectiveness and cost of gene therapy to allogeneic hematopoietic stem cell transplantation (HSCT) in patients with major beta-thalassemia. Gene therapy was found to be associated with fewer complications and hospital admissions over 2 years, but was nearly twice as costly.
A small clinical trial shows that gene therapy can safely correct the immune systems of infants with X-SCID, a rare life-threatening disorder. The treatment involves inserting a normal copy of the IL2RG gene into the patient's blood-forming stem cells.
Infants with X-linked severe combined immunodeficiency (SCID-X1) have been cured of the disease using gene therapy developed at St. Jude Children's Research Hospital. The treatment produced functional immune cells, including T cells and B cells, in all but one patient.
Researchers found that Hirschsprung disease shares common genetic mechanisms with complex disorders like schizophrenia and autism. The study identified key genes, cellular mechanisms, and molecular targets for personalized treatment.