Articles tagged with Gene Therapy
Duke researchers have developed a new method to precisely control gene activity by chemically manipulating proteins that package DNA. This technology allows for the activation of specific gene promoters and enhancers, which could provide a new avenue for gene therapies and guiding stem cell differentiation.
Researchers mapped genetic changes in tumor samples from 10 men with prostate cancer, revealing a common ancestral cell and shared gene faults. The study provides insight into how prostate cancer spreads and evolves, potentially leading to new treatment targets and personalized approaches.
Researchers at ETH Zurich have developed a novel biotechnological solution to treat erectile dysfunction by triggering reliable erections with gene therapy and blue light exposure. The system works similarly across all mammals, including humans, and is expected to have few side effects.
A potential treatment for myotonic muscular dystrophy has been identified, using an experimental drug that improves muscle function and reduces symptoms in mice. The therapy targets excessive activity of a cellular protein called TWEAK and its receptor Fn14, which correlates with disease severity.
Researchers have successfully treated dogs with hemophilia using a mutant human protein with unusually high blood-clotting power. The treatment eliminated pre-existing antibodies and reversed the bleeding disorder in two years without causing thrombosis or immune reactions.
Researchers at the University of Birmingham have developed a method to genetically engineer immune cells to combat Nasopharyngeal carcinoma (NPC), a third most common cancer in Chinese males. The engineered T-cells use Epstein-Barr virus presence to fight NPC, offering a promising new treatment approach.
Scientists have developed a new mechanism for engineering traits governed by multiple genes, paving the way for personalized gene therapies and regenerative medicine. The approach uses the Cas9 protein to activate specific genes, allowing for precise control over multiple genes and potentially treating diseases.
Research suggests statin therapy provides greatest benefit to patients with highest genetic risk of heart attack. Genetic analysis assesses heart attack risk independently of traditional factors, identifying those at higher baseline risk who reap greater benefits from therapy. The study's findings have implications for precision medici...
Researchers identified a genetic risk score that predicts cardiovascular death and heart attack risk, with individuals in the highest genetic risk group experiencing a significant reduction in cardiovascular risk with statin therapy. The study found a three-fold increase in absolute risk reduction in those with high genetic risk.
Researchers at Boston University School of Medicine identified a specific genetic signal, miR-10b-5p, that strongly correlates with disease severity and extent of neuronal death in Huntington's disease. This discovery may provide a faster and more effective way to determine the effectiveness of treatments.
Researchers have devised a clonal strategy to ensure adult epidermal stem cells meet the highest safety criteria before use in treatments. The approach involves cultivating, genetically modifying and testing individual cells to guarantee their viability and safety.
Researchers at Indiana University identified a unique gene signature in pancreatic cancer patients who can benefit from targeted therapies cutting off the growth pathways fed by abnormal blood vessels. This finding suggests that these patients may benefit from precision medicine approaches.
Researchers at UT Dallas have created a novel gene-delivery system that shuts down after delivering a gene, offering a potential new strategy for treating diseases. The approach sidesteps health problems associated with permanent gene alteration.
Pioneers in gene transfer technology, Drs. Mulligan and Miller developed the first DNA-based vectors to stably transfect cells with selectable markers. Their work laid the foundation for retroviral vector-based gene therapy strategies, leading to clinical testing and potential treatment of genetic diseases.
Researchers at Johns Hopkins Medicine have developed a new nanoparticle-based gene therapy that effectively kills brain cancer cells in rats and lengthens their survival. The treatment uses biodegradable nanoparticles filled with genes for an enzyme that turns a compound into a potent killer of cancer cells.
A recent study reveals that sea slugs can absorb genes from the algae they eat, enabling them to photosynthesize and survive for months on sunlight. This natural process has implications for gene therapy and rapid evolution in multicellular species.
Researchers at the University of Maryland have discovered a new mechanism of inheritance in roundworms that could revolutionize our understanding of evolution and disease treatment. The team found that genes can be silenced through a process called RNA interference, and this silencing can persist for multiple generations.
A study found that multiplexed genetic screening for EGFR and ALK gene rearrangements followed by molecularly-guided treatment is cost-effective compared to standard chemotherapy. The approach offers better outcomes in terms of quality-adjusted life years gained and life-years, making it a valuable option for patients with NSCLC.
Researchers at Henry Ford Hospital identified a gene that may contribute to keloid scar development. The study found altered AHNAK gene expression in keloid tissue, suggesting a potential target for novel treatments.
Researchers have identified a critical safety parameter related to AAV gene therapy, which can increase liver cancer risk. They found that modifying the vector to avoid targeting specific genes reduces cancer incidence, paving the way for safer gene therapies.
Scientists have identified several genetic mutations in the Ebola virus that could render sequence-based treatments ineffective. The study found 10 new mutations that may interfere with monoclonal antibody, siRNA, and PMO drugs, highlighting the need to consider genetic drift when developing potential therapeutics.
A study found that AAV vector integration and microRNA expression influenced liver cancer development after gene therapy. The study highlights the importance of considering vector design features when designing AAV vectors for gene therapy.
An international research team identified a critical gene linked to childhood blindness and vision loss. The study reveals that mutations in this gene affect phospholipid metabolism, leading to photoreceptor death and vision impairment.
Researchers have identified distinct gene pathways altered by infection and diet in vascular tissue, suggesting future therapies may need to be tailored. This discovery sheds light on the complex development of atherosclerosis and its association with infections like Porphyromonas gingivalis and Chlamydia pneumoniae.
Researchers at CNIO have developed a gene therapy that reactivates the telomerase gene only in the heart of adult mice, resulting in increased survival rates and regeneration of cardiomyocytes. This study provides proof-of-concept for treating chronic and acute heart failure and opens new avenues for age-related diseases.
Katherine High, Amit Nathwani, Arthur Nienhuis, and Andrew Davidoff honored for their groundbreaking research on gene therapy for hemophilia B. They developed successful clinical applications using adeno-associated virus vectors and demonstrated stable therapeutic expression of the clotting factor.
James Ingle, a renowned breast cancer expert at Mayo Clinic, has been awarded the 2014 William L. McGuire Memorial Lecture Award for his groundbreaking research on pharmacogenomics in breast cancer treatment. His work aims to develop personalized approaches to prevent breast cancer in high-risk women by identifying genetic biomarkers t...
A new genetic therapy has restored light response to the retinas of blind mice and dogs, offering hope for future clinical trials in humans. The therapy uses a virus to insert a gene into normally blind cells, followed by photoswitches that activate the cells when exposed to light.
Researchers found that a specific protein's conformational defect causes Autosomal Dominant Lateral Temporal Lobe Epilepsy, a form of familial epilepsy. Treatment with chemical chaperone LGI1 mutant protein was shown to ameliorate seizure susceptibility in mice.
Researchers have discovered novel combinations of targeted therapies and chemotherapy regimens that improve survival outcomes for patients with historically poor-prognosis leukemia. These findings, presented at the American Society of Hematology Annual Meeting, offer new hope for elderly and aggressive genetic mutation patients.
Scientists have made a breakthrough in treating epidermolysis bullosa by using induced pluripotent stem cells to grow back healthy skin cells. The new technique, called therapeutic reprogramming, was tested on mice and showed promising results.
Researchers have discovered a gene variant, PHACTR1, that reduces the risk of cervical artery dissection and other related conditions. This finding could lead to new treatment strategies for stroke prevention in young adults.
A UCL-led team of scientists has uncovered the structure of pores found in cell nuclei, revealing how they selectively block certain molecules from entering to protect genetic material and normal cell functions. The discovery could lead to the development of new antiviral drugs and better delivery mechanisms for gene therapy.
Researchers use genetically engineered pig models to study human diseases and identify new drug targets for cancer, cardiovascular diseases, diabetes, neurodegenerative diseases, ophthalmology, and xenotransplantation. These models enable the development of novel cell therapies to restore diseased tissues and organs.
Researchers have found four novel loci associated with eosinophilic esophagitis, a severe food allergy. The study suggests that genetic factors play key roles in the disease and may offer attractive targets for therapy. Understanding these biological networks could lead to tailored approaches for treatment.
Researchers have identified specific genetic mutations in melanoma tumors that predict effective responses to a groundbreaking immunotherapy. The discovery, published in the New England Journal of Medicine, could lead to more targeted and personalized cancer treatments, including tailored therapies for patients with diverse tumor genomes.
Researchers developed a gene therapy that boosts Factor IX levels in men with severe hemophilia B, resulting in significant reductions of 90-94% in spontaneous bleeding episodes. The treatment has enabled patients to adopt more active lifestyles without needing frequent clotting factor injections.
Researchers established induced pluripotent stem (iPS) cells from Werner syndrome fibroblasts, which can be used for drug discovery and gene therapy. The iPS cells have recovered telomeric abnormalities and similar expression levels of aging-related genes as normal iPS cells.
A promising anti-cancer approach targeting Malt1 protein's protease function in lymphoma cells may also cause destructive autoimmune inflammation when its other functions are preserved. Researchers discovered this unexpected side effect using an animal model, emphasizing the need for new alternatives to treat lymphomas.
A new gene therapy treatment has demonstrated effectiveness in a canine model of DMD, skipping exons to produce functional dystrophin protein. The treatment was well-tolerated and resulted in high levels of dystrophin expression, with muscle strength increasing at higher doses.
A 47-year-old woman with intrahepatic cholangiocarcinoma received a uniquely personalized treatment based on her genetic profile, achieving significant tumour shrinkage and symptom improvement. The use of next-generation sequencing revealed a rare BRAF mutation, which was targeted by dabrafenib and trametinib therapies.
Researchers at CNIO have developed a mouse model that expresses the most common K-Ras mutation found in Noonan patients, reproducing its most representative features. The study aims to advance knowledge of the syndrome and develop specific treatments for each mutation.
Scientists discovered eight shared molecular pathways and several key driver genes in Type 2 diabetes and cardiovascular disease. These findings suggest that treating the two conditions together could be effective, with potential therapeutic targets identified.
George Dickson receives 'Scientist of the Year' award from the Muscular Dystrophy Campaign for his pioneering work on novel therapies for rare diseases like Duchenne Muscular Dystrophy. The award recognizes his dedication to researching potential treatments and cures for people with DMD and related neuromuscular conditions.
Researchers at Stanford Medicine have devised a new way to edit the genome without using enzymes or promoters, which may lead to a safer and longer-lasting treatment for genetic diseases like hemophilia. The technique successfully cured mice with hemophilia by inserting a gene for a clotting factor missing in the animals.
Researchers have identified 107 genes that contribute to the risk for autism spectrum disorder (ASD), highlighting three key pathways: synaptic function, chromatin remodeling, and transcription. These findings provide a better understanding of genetic and cellular changes in ASD and may eventually lead to potential therapies.
A research team led by NUS scientists has identified several potent inhibitors that selectively target FTO, a gene strongly linked to obesity. The discovery paves the way for the development of novel anti-obesity drugs and treatments.
Researchers developed an AAV-mediated gene therapy approach to correct hyperbilirubinemia in a mouse model of Crigler-Najjar syndrome type 1. The treatment achieved significant, long-lasting reductions in bilirubin levels, with 50-70% reductions maintained throughout the study.
A new form of gene therapy for SCID-X1 appears effective and safe, correcting the disease with a functioning immune system in seven out of eight patients. The therapy's long-term safety is still being monitored, but preliminary results suggest a reduced risk of leukemia compared to previous trials.
A modified y-retrovirus vector has been found to restore the immune systems of children with X-linked severe combined immunodeficiency, a rare and life-threatening inherited condition. The new approach is equally effective at restoring immunity and may be safer than previous gene therapy methods.
Researchers discovered that mutations in the PIK3R1 gene, particularly R348, can activate ERK and JNK signaling cascades, enabling tumor growth. Targeted therapies may need to focus on these mutant tumors, offering a potential new approach for treating endometrial and colon cancers.
Researchers describe a novel liver-directed gene therapy approach that corrects heart symptoms in a model of rare enzyme disorder MPS I. The treatment, using an AAV vector, achieves sustained serum enzyme activity and reduces glycosaminoglycan storage in tissues.
Researchers found large droplets of triglycerides within neurons of mice modeling the disease, leading to potential therapies and a new investigative strategy. The study points to triglyceride metabolism as a key factor in hereditary spastic paraplegia.
Researchers found that adding rapamycin to an immunotherapy approach strengthened the immune response against brain tumor cells, increasing the effect of new therapies. The study also showed increased memory cells, allowing the immune system to attack tumors more effectively.
The Jeffrey Modell Foundation has awarded a research grant to a Belgian laboratory led by Adrian Liston to develop a gene therapy for children suffering from IPEX syndrome. The gene therapy aims to correct the mutation responsible for the disease, offering a potential cure for this rare and fatal autoimmune disorder.
A Penn researcher and CVS Health physician suggest an alternative payment model for gene therapy, which would replace single large payments with annuity payments over a defined period. This approach could help ensure sufficient investments in expensive gene therapies by spreading out payments and linking them to evidence of continued e...
Researchers have developed a new reprogramming factor cocktail that produces high-quality induced pluripotent stem cells with fewer genetic abnormalities. The SNEL combination outperforms existing methods, such as OSKM, in terms of cell quality and efficiency.
The Pioneer Award recipients have made significant contributions to gene therapy for retinal degeneration. Their research has led to proof-of-concept studies demonstrating the feasibility of using gene therapy to repair photoreceptor defects in the eye.
A new gene therapy approach has been shown to be effective in preventing and treating botulism exposure, with a single treatment leading to prolonged production of antitoxin proteins. The therapy, known as VNA gene therapy, can protect against toxin-mediated diseases such as C. difficile and Shiga toxin-producing E. coli infections.
Researchers at the University of Pennsylvania have successfully treated lysosomal storage disease MPS I using gene therapy, reducing harmful protein accumulation and improving symptoms. The treatment involves direct gene transfer into glial and neuronal cells in the brain and spinal cord.