Articles tagged with Gene Therapy
Researchers report remarkable benefits from new therapies for bleeding and clotting disorders, including emicizumab's high effectiveness in preventing bleeds in children with hemophilia A. Additionally, a gene therapy treatment provides durable improvement in clotting factor levels and long-term protection from bleeds.
Researchers announce innovative tools and therapies for hard-to-treat blood disorders, including gene editing platform CRISPR/Cas9. Interim results from a Phase I/II clinical study show restoration of all immune cell types in infants with X-linked severe combined immunodeficiency.
Early evidence suggests that a gene therapy developed at St. Jude improves immune systems in infants as young as 2 months old with XSCID, offering broad protection and reducing the need for protective isolation. The treatment has been shown to be well-tolerated and effective in restoring immune function.
The Phase 1/2 clinical trial showed that SPK-9001 reduced annualized bleeding rate by 97% and factor IX concentrate use by 99% in participants with hemophilia B. The data suggest a one-time infusion of SPK-9001 may sustain factor IX activity levels, reducing bleeding episodes.
Researchers at Salk Institute create a new version of CRISPR/Cas9 that can activate genes without creating DNA breaks, potentially treating diseases such as diabetes and muscular dystrophy. The technology operates epigenetically, influencing gene activity without changing the DNA sequence.
Researchers found a shift in cytokine profiles of mice with FVIII inhibitor development, including increased levels of pro-inflammatory cytokines. A negative correlation was also observed between risk factors for FVIII inhibitor development and age at gene therapy administration.
A team of researchers from the Broad Institute of MIT and Harvard has developed a new compound that can protect kidney cells from death and restore kidney function in multiple animal models of progressive kidney disease. The compound, called AC1903, works by blocking a damaging feedback loop involving the protein TRPC5.
A new gene therapy has successfully produced sustained levels of clotting factor in adult hemophilia B patients, eliminating the need for regular infusions. The treatment, which uses a highly active variant of the clotting factor, achieved significant clinical benefits with no adverse effects.
A new gene therapy transplantation technique could improve treatment of neurodegenerative diseases by generating genetically engineered microglia-like cells in the brain. This technique has been tested on an experimental model for a metabolic disease and may have future therapeutic applications for other neurodegenerative diseases.
Scientists have made a breakthrough in treating neurodegenerative disorders and lysosomal storage diseases by transplanting hematopoietic stem cells directly into the brain, achieving therapeutic benefits faster than traditional methods. This innovative technique could pave the way for new treatments for Parkinson's, Alzheimer's, and o...
Researchers found seven genes with altered expression in melanoma patients, which could serve as biomarkers for diagnosis and therapeutic targets. The synthetic compound DM-1 induces cell death by increasing the expression of TOP-1 and ADK genes.
The article examines the persistence of continuous electronic fetal monitoring as a standard of care, despite evidence that it increases risks. It also discusses the lack of mechanisms for ensuring medical professionals stay current with best practices and the role of malpractice law in setting boundaries on acceptable interventions.
Researchers designed new therapeutic DNA aptamers with diverse side chains to enhance interaction with targets. The study found that the hydrophobicity of side chains affects clearance from the bloodstream, providing a guide for designing better aptamers.
Researchers developed a novel gene therapy that lasts for over eight months, reducing methamphetamine in the brain and its stimulant effects. The therapy generates antibodies against meth, preventing it from reaching the brain.
A new gene therapy has shown significant improvements in vision for patients with Leber congenital amaurosis, a rare inherited retinal disease. The treatment, currently under FDA review, allows patients to navigate mazes and experience improved light sensitivity and peripheral vision.
A boy suffering from extensive skin damage due to epidermolysis bullosa was successfully treated with genetically modified stem cell transplants. The treatment resulted in the growth of high-quality, stress-resistant skin, allowing him to attend school and participate in social life.
A Phase 1 clinical trial has demonstrated encouraging data for gene replacement therapy in patients with spinal muscular atrophy type 1 (SMA1). The study found that high-dose gene therapy improved motor function and reduced the need for supportive care in patients treated with a single intravenous infusion.
A study published in Annals of Oncology has identified genetic markers that may predict resistance to a specific therapy used for treating advanced prostate cancer. The researchers found that understanding the genetic markers of tumors can help healthcare providers match a therapy that is likely to succeed on the first try.
Researchers at Case Western Reserve University School of Medicine have developed a gene therapy that can prevent the progression of hearing loss and preserve hearing in people with Usher syndrome type III, a form of hereditary hearing loss linked to defects in sensory hair cells.
A groundbreaking study has found that injecting DNA into injured horse tendons and ligaments can cure lameness within two to three weeks. The gene therapy technology also showed no adverse side effects, with the horses returning to full health after just two months.
German researchers have made significant advancements in human gene therapy, including virotherapy capable of destroying tumor cells and engineered hematopoietic stem cell delivery systems. These innovations hold promise for treating immunodeficiencies and genetic diseases.
Researchers have identified 13 new genetic risk variants for restless legs syndrome, a condition affecting up to one in ten people of European ancestry. The findings suggest that the condition may be related to neurodevelopmental disorders and offer clues for developing new treatments, including drugs like thalidomide.
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 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.
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.
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.
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.
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 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.
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.
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.