Articles tagged with Gene Therapy
A group of inflammatory proteins called 'IL-36' play a significant role in the development of psoriasis and can be blocked without posing a risk to patients. The findings open up the possibility for new drugs to tackle this debilitating condition.
M344 altered key gene expression in a cellular model of AD, preventing cognitive decline and improving memory. In mouse models, low-dose administration of M344 prevented cognitive decline and improved learning and memory.
A recent study has identified 150 genetic drivers of diffuse large B cell lymphoma, a common form of blood cancer. The research, led by Duke Cancer Institute scientists, found correlations between specific genes and treatment responses, opening up new avenues for targeted therapies.
A new study successfully uses a shortened version of the CEP290 gene to treat Leber congenital amaurosis type 10, a rare genetic disorder causing blindness. The treatment shows significant improvement in photoreceptor survival and function.
A Swedish national adoption study found that children of divorced parents are more likely to get divorced due to genetic factors, not environmental influences. The study suggests that targeting basic personality traits linked to divorce may be a better approach for therapists.
A new study in The FASEB Journal reveals that over-expression of the GTRAP3-18 gene is linked to obesity in humans. Inhibiting its interaction with POMC might be a strategy for treating leptin/insulin resistance in patients with obesity and type 2 diabetes.
Researchers report that a gene therapy to treat cerebral adrenoleukodystrophy has effectively stabilized the disease's progression in 88 percent of patients. Fifteen out of 17 patients had stable neurologic functioning more than two years after receiving the treatment.
Researchers used gene therapy to express melanopsin in mouse retinas, restoring visual function and improving pupil constriction. The treatment was effective for up to 15 months, showing enhanced visual responses and light avoidance behaviors.
A new study found that customizing warfarin dosing to a patient's genetic and clinical profile reduces adverse outcomes compared to clinically-guided dosing. The study involved 1,650 patients undergoing hip or knee replacement, who were randomly assigned to either genotype-guided or clinically-guided dosing.
Researchers used gene therapy to improve heart function in a large animal model, finding a 25% improvement in left ventricular function and a 20% improvement in left atrial function. The treatment also reduced heart size by 10%.
Researchers at Institut Pasteur successfully restored hearing and balance in a mouse model of USH1G syndrome using gene therapy, offering new hope for the treatment of hereditary deafness. The study demonstrates that inner ear defects caused by mutations in the USH1G gene can be reversed even after birth with durable efficacy.
Researchers developed a novel gene delivery approach using scaffold/matrix attachment region (S/MAR) to enhance long-term transgene expression in proliferating cells. The modified vectors were able to establish colonies and maintain stable transgene expression in HeLa cells.
A potent gene immunotherapy strategy induces robust immune tolerance in mice with multiple sclerosis, reversing symptoms and stabilizing brain function. The treatment, using a viral vector to deliver myelin sheath protein, protects against disease progression and restores mobility.
Researchers used gene therapy to stop the immune response that causes multiple sclerosis in mouse models, producing near-complete remission. The treatment combined a brain-protein gene with an existing medication, showing significant potential for treating multiple sclerosis and other autoimmune disorders.
A special issue in the Journal of Dental Research explores recent scientific and technical advances in craniofacial development and genetics. The discoveries reported could improve diagnosis and treatment of challenging craniofacial and dental anomalies.
Researchers identified two cytokines and genetic markers associated with progressive MS, which may enable the development of a treatment to prevent disease progression. A simple genetic test could identify patients at risk, and a medication is already in development.
A new study uses gene transfer to block the expression of an enzyme that breaks down alcohol, leading to increased acetaldehyde levels and potential treatment for alcoholism. The approach has strong proof-of-concept data and bodes well for future development of a genetic therapy.
Researchers identified macrophage migration inhibitory factor (MIF) and its related protein D-dopachrome tautomerase as closely related cytokines associated with progressive MS. A genetic test could identify patients at risk of developing the disease, accelerating drug development for precision medicine.
Scientists at Oregon State University developed a new assay to study the otoferlin protein, essential for hearing. They found a truncated version of otoferlin that can function in sound encoding and validated a method for characterizing large membrane proteins.
Clinical evidence supports the use of mesenchymal stem cell transplantation to treat Crohn's disease-related fistulas, offering a safe and effective approach. The unique advantages of MSCs include ease of collection, low immunogenicity, and immunoregulatory activity.
Researchers at Brigham and Women's Hospital discovered a rare genetic mutation in the ALK gene that causes peritoneal mesothelioma in young women with no history of asbestos or radiation therapy. Targeted therapy using FDA-approved drugs may be effective in treating these patients.
A genetic mutation in the CLPX gene has been identified as a potential cause of erythropoietic protoporphyria (EPP), a form of porphyria that may have inspired vampire folklore. The discovery highlights the complex genetic network underlying heme metabolism and holds promise for future therapies.
Neuroscientists at the University of Nevada, Reno identified a genetic mechanism in fruit flies that disrupts brain pathways connecting the left and right hemispheres, separately linked to autism. The study suggests that the human gene PRRG4 may play a role in regulating nerve fiber guidance and synapse formation.
A new clinical investigation is testing whether genetic screening can identify patients with a specific mutation who are the best candidates for deep brain stimulation surgery. The study aims to determine how this genetic information can inform surgical decisions and potentially improve outcomes for Parkinson's disease patients.
Researchers at Fred Hutchinson Cancer Center developed a nanoparticle delivery system to extend the therapeutic potential of messenger RNA, delivering gene changes to targeted cells. The 'hit-and-run' genetic programming approach can be easily scaled up for various disease applications.
The FDA has approved Kymriah, a CAR T gene therapy drug, for pediatric and young adult patients with ALL, marking a major milestone in cancer treatment. The therapy showed an exceptional 83 percent success remission rate in clinical trials.
Researchers used gene therapy with LeXis, a 'junk DNA' suppressor of cholesterol, to reduce plaque in mice with familial hypercholesterolemia. The treatment lowered cholesterol and blockages in arteries, and appeared to reduce fat build-up in liver cells.
A team led by Dr. Rena D'Souza aims to identify the relationship between PAX9 and WNT pathways in permanent tooth formation. The goal is to develop new therapies that reestablish normal interaction between genes and pathways to restore tooth development.
Scientists explore gene therapy for injured peripheral nerves, aiming to improve functional recovery after microsurgical repair. The approach involves modifying a directional cue for nerve cells to promote fast regeneration and functional recovery.
Researchers hope IBM's Watson will help people with cancer by providing detailed information on genetic mutations and targeted therapies. With Watson, pathologists can analyze multiple genes simultaneously, including those that are not currently tested, to provide more personalized care to patients.
Researchers at the University of Chicago have developed a novel gene therapy approach that uses skin transplants to treat type-2 diabetes and obesity. The method uses CRISPR technology to deliver a hormone that stimulates insulin production, reducing blood glucose levels and promoting weight loss.
The Massachusetts General Hospital team, led by J. Keith Joung, is developing more sensitive methods of measuring and controlling on-target gene-editing activity and detecting off-target mutations induced by CRISPR-Cas9 technology.
Researchers developed a gene therapy using microdystrophin to treat Duchenne muscular dystrophy in dogs. The treatment restored muscle function and stabilized clinical symptoms for over 2 years. This breakthrough could lead to a new treatment for children with the disease.
A comprehensive study published in Nature Communications reveals that lean body mass is highly heritable and can be attributed to specific genes. The research may lead to the development of future therapies to prevent the loss of lean mass and associated disabilities such as sarcopenia.
The Phase 3 clinical trial data showed statistically significant and clinically meaningful improvements in functional vision and visual field in participants with RPE65-mediated inherited retinal disease. Participants demonstrated marked gains in full-field light sensitivity and peripheral vision.
The FDA advisory committee voted unanimously to recommend approval of Novartis' CAR-T therapy Tisagenlecleucel, demonstrating impressive results in hard-to-treat leukemia patients. ACGT's funding played a crucial role in advancing the pioneering treatment.
A proof-of-concept study demonstrates the potential of nanoparticles in delivering molecules that target specific genetic markers in glioblastoma brain tumors. The therapy stops tumor growth and extends survival when administered continuously through an implanted drug infusion pump.
Researchers have developed novel secreted reporter proteins that can be detected for several months after infusion into the liver, demonstrating potential for monitoring therapeutic gene delivery and ongoing expression. The findings show promise for scaling up this method to humans.
Researchers have successfully delivered a gene therapy to the brains of mice with mucopolysaccharidosis type 1, preventing neurologic deficits. The noninvasive intranasal approach shows promise for treating genetic disorders affecting the brain.
Researchers at Wayne State University are investigating how genetic factors impact the benefits of exercise, using a fruit fly model to identify novel targets for therapeutic pharmaceuticals. The study aims to understand how exercise adaptations can be induced in patients who cannot perform regular physical activity.
A study has characterized the mouse genome, revealing new insights into rare diseases and accelerating the development of new treatments. The research identified 360 new disease models and 28,406 new descriptions of genes' effects on mouse biology and disease.
A study has identified a common gene mutation linked to retinitis pigmentosa in Hispanic families from the Southwestern US, with over 70 genes now known to cause the disease. The researchers found that a dominant mutation in the arrestin-1 gene is responsible for 36% of cases, offering hope for future treatments and therapies.
A new study published in Cell suggests that virtually any gene can influence disease, with peripheral genes having tiny but significant effects. The 'omnigenic model' proposes a more nuanced understanding of genetic variation and its role in disease.
Researchers found that artificial viruses can infect brain cells and surrounding tissues beyond the injection site, influencing the immune response. The study's findings could improve the selection of suitable viral 'gene transporters' for custom therapies, offering a glimmer of hope for patients with Alzheimer's and Parkinson's.
Researchers have discovered a solution to evade the immune system, allowing more patients to participate in gene therapy clinical trials. The altered virus can slip past neutralizing antibodies, making it possible for up to 70% of patients previously ineligible to join trials.
Researchers at German Cancer Research Center (DKFZ) have discovered mysterious gene transcripts that arise in treated cancer cells. These transcripts, originating from ancient viral elements, may be used as biomarkers to monitor the effectiveness of epigenetic therapy.
A new study found that oral prednisone reduced immune responses to AAV-based gene delivery, resulting in a 60% decrease in cytotoxic T cell infiltrates. The treatment also increased PD-L2 levels, which induce programmed T-cell death, suggesting a potential therapeutic benefit for gene therapy.
Researchers conducted a study assessing stem cell transplantation for patients with a rare 'gain of function' STAT1 gene mutation. The treatment showed encouraging results, with five patients being completely cured and disease-free, but the overall success rate was disappointing at 40%. Experts propose adjusting treatment parameters, s...
A new gene therapy has been shown to preserve muscle strength and neurologic function in dogs with myotubular myopathy, a type of muscular dystrophy. The therapy, which involves replacing the mutated MTM1 gene, has provided long-lasting benefits over 4 years.
Recent clinical trials using different gene transfer approaches have shown acceptable risk and potential for treating heart failure with reduced ejection fraction. Despite mixed results in terms of efficacy, the data support further clinical development of this therapeutic strategy.
A French study of 1,944 patients with advanced cancer found that comprehensive genomic profiling can guide treatment choices and lead to higher survival rates. The study's results suggest that routine genomic testing may not benefit all patients but offers hope for personalized medicine.
A study found that combining therapies targeting polyADP ribose polymerase (PARP) and mitogen-activated protein kinase (MEK) inhibitors showed promise in treating RAS-mutant cancers. The combination therapy was effective in multiple tumor models, regardless of mutations in tumor suppressor genes.
Researchers in China have made a breakthrough in developing a new approach to treat premature ovarian failure using female germline stem cells. The study found that mice with transplanted egg-making stem cells produced healthy pups without genetic malfunctions, opening up avenues for investigating human oogenesis in vitro.
Researchers have developed a novel gene therapy method that utilizes microbubbles and ultrasound to promote bone growth and healing in nonunion fractures. The technique, which involves injecting genetic material for a bone growth factor into the affected area, has been shown to completely heal broken bones in pigs within eight weeks.
Researchers have shown that an experimental gene therapy is safe and may be effective in preserving vision in people with wet age-related macular degeneration. The treatment involves injecting a virus containing a therapeutic gene into the eye, which produces a protein to prevent abnormal blood vessel growth.
Researchers have discovered a more favorable virus species for delivering genes, as its surface structure avoids liver toxicity. This could enable the use of viruses in gene therapies and vaccine development.
Researchers at Case Western Reserve University developed a gene-carrying nanoparticle that delivers healthy RPE65 genes to photoreceptor cells, preventing vision loss in mice with Leber congenital amaurosis. The therapy's success indicates promise for treating other inherited visual disorders.
The study discovered genetically coupled promoters in viral-host networks, presenting an additional layer of regulation between virus and host. The findings reveal a mechanism for synchronizing gene expression in synthetic gene circuitry and provide novel design principles to reverse-engineer viral circuitry.
A new review discusses how gene therapy delivered via adeno-associated viruses (AAV) can trigger an immune response in T cells, leading to activation and exhaustion. The review highlights the variability in individual patients' immune responses to AAV vectors and potential strategies to suppress these reactions.
A growing number of preclinical studies suggest that rAAV-mediated gene therapy may cause insertional mutagenesis and increase the risk of hepatocellular carcinoma. Research emphasizes the need for careful reconsideration of potential genotoxicity risks in human clinical applications.