Articles tagged with Gene Therapy
Researchers at Mayo Clinic have developed a potential test for Machado-Joseph disease, a rare neurodegenerative disorder with no cure. The new test can quantify mutant protein levels in human biofluids, which may help evaluate the effectiveness of therapies aimed at reducing protein accumulation.
A new gene therapy using a novel light-sensing protein has restored significant retinal function and vision in blind mice. The therapy involves attaching the MCO1 opsin to retina bipolar cells using gene therapy, allowing treated mice to navigate mazes and detect changes in motion faster than untreated mice.
A UCI-led study demonstrates the therapeutic potential of base editing for treating inherited ocular diseases, restoring visual function to near-normal levels. The new CRISPR technology overcomes previous barriers, enabling precise and predictable correction of point mutations.
A new study has identified three gene variants that may help predict which children with eosinophilic esophagitis (EoE) are likely to respond to proton pump inhibitor (PPI) medication therapy. The research found that children carrying these variants are at increased risk of relapse after one year of PPI maintenance therapy.
Researchers create an artificial C-to-U conversion system using APOBEC1, allowing for the restoration of mutated genes and potentially treating genetic disorders. The system was tested on blue fluorescent protein (BFP) RNA with a 199T>C mutation, showing high editing efficiency.
University of Texas at Dallas researchers have identified the source of fluctuations in gene expression between identical cells, finding that transcription is the primary driver of cellular noise. This discovery has significant implications for developing targeted therapies and gene therapies.
Researchers at Kanazawa University discovered that thymoquinone selectively kills prostate cancer cells with a SUCLA2 gene deletion. This finding offers a promising therapeutic option for advanced prostate cancer patients who have developed resistance to hormone therapy.
A young woman with kaposiform lymphangiomatosis was treated with a MEK inhibitor, resulting in complete resolution of symptoms and remodeling of her lymphatic system within months. The treatment showed promise for other patients with lymphatic disorders.
Research reveals genetic differences in fat storage and formation affecting disease susceptibility in men and women. Six genes were identified as potential therapeutic targets for distinct outcomes in each sex.
Researchers identified 182 genes that regulate interaction between cancer and immune T cells, which could be harnessed for effective immunotherapy across different tumour types. The study also revealed the need for new therapy to account for genetic mutations in cancer cells that can make disease worse in response to treatment.
Researchers successfully used gene therapy to overcome cardiac effects of Friedreich's ataxia in a mouse model, achieving exercise performance similar to healthy littermates. The treatment delivered the frataxin gene via adeno-associated virus (AAV) and showed promising results.
Scientists accelerated vision restoration by visualizing photoreceptors' response to light in the living eye. Researchers modified OCT technology to detect minute changes, enabling high-resolution visualization of cone photoreceptors and potential therapies like stem cells or gene therapy.
Researchers at Children's Medical Research Institute have discovered a reason behind the low success rate of gene therapy targeting liver diseases using Adeno-associated virus 2 (AAV2). The team found that AAV2 binds too tightly to its attachment receptor, heparan sulfate proteoglycans, which leads to the vector getting
Capmatinib shows high response rate (68%) in first-line treatment for NSCLC patients with MET exon 14 skipping mutations. Patients with lower MET amplification levels have limited effectiveness with the drug.
Researchers have found that specific DNA variants can affect the response to interferon-alpha-based treatments, leading to personalized therapy options. The discovery may improve treatment outcomes for patients with polycythaemia vera, a rare chronic malignant blood disease.
A new trial suggests that children with asthma may benefit from personalized treatment based on genetic differences. The study found that children with two copies of a specific gene variant experienced an average improvement in quality of life score of 0.42.
Researchers created a system that uses CRISPR to briefly suppress genes related to AAV antibody production, allowing the virus to deliver its cargo unimpeded. The study shows promise for improving gene therapy's effectiveness and preventing or treating sepsis in mice.
Researchers have developed a novel gene therapy approach that activates genes with similar functions to compensate for the primary defect in retinitis pigmentosa. This method has shown promise in slowing down retinal degeneration and improving retinal function without side effects.
A meta-analysis of NHP studies reveals that AAV gene therapy often causes DRG pathology with no clinical effects, prompting preclinical safety evaluations before clinical trials. The study's findings have the potential to streamline the development process for new vectors.
A study by University of Pennsylvania researchers describes an effective gene therapy for Krabbe disease in dogs, which has shown promising results with no significant symptoms after four years. The treatment approach, using the AAV9 vector, has also been found to positively affect both central and peripheral nervous systems.
A new clinical trial, TAILOR-PCI, found that genetic testing can identify patients who may benefit from alternative antiplatelet therapy. The study showed a statistically significant 40% reduction in blood clotting events in patients with the CYP2C19 gene variation who received genotype-guided treatment.
A new study published in Pediatrics reports positive safety and early outcome data from 21 children with spinal muscular atrophy treated with gene therapy. The treatment, onasemnogene abeparvovec-xioi, is shown to be effective through age 2 years with proper screening and monitoring, but requires closer attention for potential liver is...
Researchers have discovered new genes that play a crucial role in determining the success of radiotherapy in cancer treatment. These genes, which encode for repair mechanisms used by cancer cells, can be missing or dysfunctional in some patients, making them more likely to respond to radiation therapy.
Scientists have engineered a tiny therapeutic delivery system using lipid-based nanoparticles that safely solve genetic problems in mice. The treatment successfully produced a protein to treat hemophilia and reduced gene activity to lower cholesterol levels, offering hope for targeted therapies.
Researchers at the University of Tokyo have shown that a treatment enhancing neuromuscular junction (NMJ) formation can improve motor function and muscle strength in aged mice. The study's findings suggest that this approach may be effective in treating age-related motor impairment and muscular weakness.
A McGill-led study reveals that suppressing the OSMR gene can improve radiation therapy effectiveness and expand lifespan in preclinical mouse models. Glioblastoma's resistance to therapy is overcome by starving cancer stem cells with energy production halted.
A targeted therapy called rucaparib has been successfully used to treat men with advanced prostate cancer who have mutated BRCA1/BRCA2 genes. The treatment resulted in a 41% objective response rate and improvements in prostate-specific antigen levels for over half of the patients.
Researchers developed novel variants of adeno-associated viral (AAV) capsids with improved transduction properties in the mouse retina and cornea. The efficient gene delivery of these variants was confirmed in non-human primate tissue, adding to their potential use in treating human ocular diseases.
A Singapore team identified a mutation in the PD-L1 gene as a reliable biomarker for patients with natural-killer T-cell lymphoma who are likely to respond positively to anti-PD-1 therapy. The study found an overall response rate of 47% and complete responses in 7 out of 19 patients.
Researchers at CNIO develop effective therapy for mice with age-related pulmonary fibrosis, activating telomerase production to prevent disease development. The treatment, a gene therapy, also successfully reversed fibrosis in mice without genetic alterations.
Researchers have discovered a promising new treatment strategy for patients with prion disease by reducing levels of prion protein. This approach has been shown to significantly extend the survival of lab animals and even reverse disease markers after toxic clumps have formed in the brain.
A breakthrough study using gene therapy to target the inner retina has prevented blindness in a mouse model of CLN3 Batten disease. The treatment led to significant survival of bipolar cells and preserved retinal function, according to researchers.
Researchers developed a nanomedicine-based strategy for chemo-immunotherapy that eradicated PTEN-negative glioblastoma cells. The combination of epirubicin-encapsulating nano-micelles and immune checkpoint inhibitors increased tumor-infiltrating T cells and reduced immunosuppressive cells, effectively killing cancer cells.
Dr. Sanjana's research uses high-throughput genome engineering technologies to identify genes that can boost the effectiveness of CAR T-cell therapy for pancreatic ductal adenocarcinoma. The project aims to overcome immunosuppression in pancreatic cancer and potentially lead to improved immunotherapies.
Researchers at the University of Pennsylvania have identified a new genetic cause of Charcot-Marie-Tooth disease, a debilitating neurodegenerative disorder that affects nearly one in two thousand people. The discovery sheds light on the genetic underpinnings of the disease and may hold answers to new gene therapies.
A new therapeutic approach for CLN3 Batten disease has been developed by a team led by Rosalind Franklin University researcher Michelle Hastings. The treatment uses antisense oligonucleotides to target the mutated gene causing the disease, showing promise in rodent models and human cells cultured from patients.
Researchers at the Wellcome Sanger Institute have engineered a new mouse model to study glioblastoma, the most aggressive type of brain cancer. The study identified over 200 genes that contribute to the development and growth of glioblastoma, providing potential new drug targets.
A study has identified a genetic synergy that can cause cancer cell death, which can be replicated by a drug-like molecule and exploited for therapy. The findings suggest that FEN1, a gene involved in DNA replication and repair, has synthetic lethal interactions with BRCA-mutant cells.
A team of researchers used a mouse model to test the best therapeutic option for a type of relapse sarcoma. The study found that two treatment regimens administered to a patient with lung metastasis helped extend their disease-free period to 46 months.
A new study has identified genes more frequently altered in prostate tumors from men of African ancestry compared to other racial groups. The research found no significant differences in frequencies of mutations in genes important for current prostate cancer therapies, but suggests that precision medicine approaches can be effective if...
Researchers successfully deliver gene therapy using nanoparticles to inhibit abnormal blood vessel growth in rats and mice eyes, providing evidence for treating wet age-related macular degeneration and inherited retinal diseases.
A recent report of two children's deaths in a gene therapy trial for X-linked myotubular myopathy has sparked concerns over the safety of gene therapy vectors. The editorial emphasizes the need for iterative development and cooperation among scientists to ensure safe and effective treatments for rare genetic diseases.
An international team identified an error in the cytochrome P450 oxidoreductase (POR) gene as the cause of aromatase deficiency, a rare metabolic disorder. This discovery enables precise therapy and prevents side effects associated with steroid supplementation.
The SARS-CoV-2 virus activates the aryl hydrocarbon receptors (AhRs) through the IDO1-kynurenine-AhR signaling pathway, leading to Systemic AhR Activation Syndrome (SAAS). This triggers a cytokine storm causing inflammation, thromboembolism, and fibrosis. Researchers propose therapies targeting downregulation of AhRs and IDO1 genes to ...
Researchers have developed a reagent for selective and safe coating of the liver sinusoidal walls to control clearance of gene therapy drugs. The coating agent improved gene transfer efficacy by 2-4 times to the myocardium and skeletal muscles, and 10 times to colorectal cancer, reducing medical costs and adverse effects.
Researchers at Kobe University have developed an exon-skipping therapy using antisense oligonucleotides to treat Alport Syndrome, a genetic kidney disease. The treatment was found to be effective in reducing urinary protein levels and suppressing kidney failure in model mice with severe mutations.
Researchers at the University of Iowa have developed a gene therapy approach to convert lab mice into models of COVID-19. This tool allows researchers to rapidly test treatments and vaccines, including poly I:C, convalescent plasma, and remdesivir, which prevented weight loss and lung disease in infected mice.
A study published in JAMA Neurology reports successful delivery of micro-dystrophin to patients with Duchenne muscular dystrophy (DMD), resulting in functional improvements. The therapy showed robust gene expression and localization, as well as improved muscle function measured by North Star Ambulatory Assessment scores.
Scientists have developed a new gene therapy approach that offers tremendous promise for treating an eye disease that leads to blindness. The collaborative team used a modified virus to deliver a normal functioning copy of the RP2 gene into 'mini retinas' containing the defective version, resulting in significant improvement.
A Phase 3 study found lumasiran to be effective in reducing 24hr urinary oxalate excretion by 65.4% and normalizing oxalate levels in patients with PH1. The treatment also showed an encouraging safety profile, with mild injection-site reactions being the most common adverse event.
Italian scientists have identified common genetic susceptibility genes linked to COVID-19 outcomes, including severe pneumonia. The study's findings suggest that these genes may determine disease progression and could aid in the development of new treatments.
A research team from Genethon has successfully inhibited the immune response induced by AAV antibodies, paving the way for repeated administration of gene therapy treatments. This breakthrough could enable treatment of rare genetic diseases and improve patient outcomes.
A Phase Ib/II trial found that adding idh1 inhibitor ivosidenib to either venetoclax or azacitidine resulted in a high complete remission rate of 78% overall, with 100% for treatment-naïve patients. The combination may ultimately lead to a new therapeutic regimen and tailored treatment options based on molecular profiles.
Nanoscope Technologies has developed a novel, non-viral gene delivery approach using low-power near-infrared lasers to target the retina. The method sensitizes cells to ambient light, improving visual response and offering a unique therapy option for dry-AMD patients.
Research identified potentially treatable genetic mutations in prostate cancer patients who did not respond to PSMA-targeted therapy. Targeted next-generation gene sequencing found mutations in six out of seven patients, including alterations in DNA damage-repair genes such as TP53, ATM, and CHEK2.
Researchers discover Foxg1's role in controlling brain electrical activity, shedding light on rare genetic diseases like Rett Syndrome and West Syndrome. Understanding this mechanism is crucial for developing future therapeutic interventions.
A study in mice suggests gene therapy can build significant muscle mass quickly and reduce the severity of osteoarthritis, even without exercise. The therapy also prevented obesity and improved cardiovascular health.
Researchers identified two previously overlooked genes as potential new therapeutic targets for cancer treatment and male infertility. RMPs play a key role in the RNA modification process, adding or removing chemical groups to the sequence, and altering the original code copied from DNA.
University of Virginia researchers are pioneering a new approach using focused ultrasound to deliver treatments directly into the brain. The technology, which can selectively open the blood-brain barrier, has shown promising results in treating conditions like Alzheimer's, epilepsy, and brain tumors.
A study identified a rare genetic variant in the ANGPTL7 gene that lowers intraocular pressure and significantly reduces the risk of glaucoma. The findings open up new therapeutic possibilities for treating this eye disease.