Articles tagged with Gene Therapy
Researchers have found that suppressing the nuclear receptor protein ROR-γ with small-molecule compounds can reduce androgen receptor levels in castration-resistant prostate cancer, stopping tumor growth. This novel approach targets the root cause of the problem - the overexpression of the AR gene and its protein.
A global study has identified hundreds of genes associated with 5 common inflammatory diseases, paving the way for new treatments. The research found that these conditions share similar genetic backgrounds and can occur together due to shared risk factors.
A new Scripps Research Institute study has identified specific genetic variations associated with increased susceptibility to bipolar disorder and other conditions. The research focuses on a gene called PDE10A, which produces proteins that regulate intracellular levels of cAMP, influencing biological processes like learning and memory.
Researchers discovered that healthy carriers of a rare respiratory disease gene have enhanced respiratory capacity and increased height, with a 10% advantage. The study may lead to new treatment approaches for physical fitness training and lung function disorders, as well as height modification in growth disorders.
Researchers have identified two genes that, when altered, promote or undermine cardiovascular health. ANGPTL4 is associated with lower triglycerides and reduced risk of coronary disease, suggesting strategies to lower triglycerides could help prevent heart attacks. Future therapeutics targeting this pathway are in development.
Researchers present innovative approaches using cell transplantation and genetic engineering to address neurodegenerative diseases, including Alzheimer's and Huntington's. Studies show promise in reducing learning deficits and alleviating peripheral neuropathic pain, offering new hope for treatment options.
Current gene sequencing methods struggle to decipher medically important genes in troublesome genome regions, leading to false positives and false negatives. Researchers call for more accurate sequencing to optimize medical care and prevent unnecessary surgeries.
Gene therapy has made significant progress in treating X-linked severe combined immunodeficiency (SCID-X1), with hematopoietic stem cells showing promise for a cure. However, ongoing challenges include improving safety and achieving long-term immune reconstitution, highlighting the need for continued research and development.
Researchers develop integrase-defective lentiviral vector to deliver chemotherapy-sensitizing gene to pancreatic tumor cells, reducing risk of insertional mutagenesis. The system enables high efficacy delivery of the gene that encodes for DCK protein to cancer cells, making them more sensitive to chemotherapeutic drug gemcitabine.
ESTEVE has developed two new investigational gene therapies, EGT-201 and EGT-301, to treat Sanfilippo B syndrome and Hunter syndrome. The treatments join a promising gene therapy platform aimed at restoring enzyme function in patients with severe and debilitating rare diseases.
A team of researchers discovered that people with Down syndrome have altered white matter insulation in their brains, affecting nerve transmission. This finding may lead to new treatments for DS patients and has implications for other developmental disabilities like autism.
Researchers have identified multiple gene therapy approaches to treat Alzheimer's disease, including boosting neuroprotection, increasing autophagy-related proteins, and regulating lipid metabolism. These strategies aim to address the underlying neuropathological changes associated with the disease.
Florida State University researchers are working on a new approach to deciphering genetic data that may lead to more targeted prostate cancer treatments. They identified altered signal pathways in prostate cancer, which could help pinpoint genetic changes that matter in cancer development and progression.
Scientists at the University of Sheffield have identified a network of genes that enables sharks to regenerate their teeth throughout their lifetime. Humans possess similar cells, but with limited tooth regeneration ability.
Researchers from Kumamoto University have identified the protein p53, which plays a crucial role in slowing down the progression of Alport syndrome. The study suggests that recovering the function of the p53 gene could help inhibit symptom progression and develop new treatment strategies for genetic diseases.
Researchers at Michigan Medicine have identified a strong interaction between KRAS and AGO2 that could contribute to its role in cancer development. The study suggests that targeting this interaction may be a potential therapy for KRAS-related cancers.
Researchers demonstrate selective gene delivery to modified upper motor neurons, showing promise for future gene replacement therapies. The study provides evidence that targets diseased cells with high specificity, laying the groundwork for effective treatment strategies.
A study found that HER2-positive lung cancer is caused by either gene amplification or mutation, rather than both. This distinction implies the need for different treatment options, including targeted therapies approved for breast cancer.
Researchers have pinpointed cell types responsible for common brain diseases, including Alzheimer's and Multiple Sclerosis. The findings suggest that developing medicines targeting microglial cells could offer hope for treating these conditions.
Researchers used CRISPR to repair a genetic mutation responsible for retinitis pigmentosa, an inherited condition causing blindness in at least 1.5 million cases worldwide. The study marks the first time researchers have replaced a defective gene associated with a sensory disease in stem cells derived from a patient's tissue.
Scientists have identified a protein called Tet3FL that acts as a 'guardian' against neurodegenerative diseases like Parkinson's. Tet3FL removes molecules called methyl groups from specific genes, keeping them active and maintaining cellular 'trash removal' processes.
Researchers discovered an abnormal gene expression in the lining of the womb that is associated with IVF failure. This finding could lead to a new test to predict treatment success and guide patient counseling, helping women understand their chances of achieving a pregnancy before starting treatment.
A University of Colorado study found distinct genetic drivers in colorectal cancer among young and old patients, with potential implications for targeted therapies.
Researchers have successfully used gene therapy to correct a rare bleeding disorder, factor VII deficiency, in dogs. The treatment showed long-term safety and efficacy, with treated dogs expressing therapeutic levels of clotting factor VII.
UCLA scientists have discovered a crucial role for the Rbfox1 gene in regulating genes that contribute to autism risk. The study highlights an untapped region in brain cells as a goldmine of drug targets for new treatments.
Researchers have identified three genetic associations that influence susceptibility to primary open angle glaucoma, a leading cause of irreversible blindness. The study found associations between TXNRD2, ATXN2, and FOXC1 genes, which may lead to the development of gene-based screening tests and therapy for glaucoma patients.
Researchers in gene therapy challenge a recent study linking adeno-associated virus 2 (AAV2) to liver cancer development, citing flawed experimental methods and data interpretation. AAV2 is found in up to 90% of humans, yet liver cancer affects only about 10/100,000 people in the US.
The Concise Guide to PHARMACOLOGY 2015/2016 provides an overview of 1,700 human drug targets, focusing on those exploited in the clinic or with future therapeutic potential. The guide combines evidence-based data with summaries of molecular targets, allowing users to rapidly gain insight into their function and comparative pharmacology.
Duke University researchers successfully treated an adult mouse model of Duchenne muscular dystrophy using CRISPR gene editing. The treatment involved delivering the gene-editing system directly to the affected tissues through a non-pathogenic carrier called adeno-associated virus, overcoming several delivery challenges.
Researchers at UTA have developed a new platform that uses ultrafast near-infrared lasers to deliver gene therapy to damaged areas of the retina, enabling vision restoration in patients with macular degeneration. The laser-based method has been shown to be more effective than traditional chemical gene delivery systems.
Researchers identified 52 genetic variations associated with AMD across 34 gene regions, offering hope for developing diagnostic tests and therapeutics. The study also found rare genetic variations in one subtype of AMD, which could explain why anti-VEGF therapy is less effective in some patients.
Researchers have identified 52 genetic variants associated with AMD, including associations between CFH and TIMP3 genes and the extracellular matrix. The study provides a framework for future studies of AMD biology and therapy development.
Researchers discovered that lung cancer in younger patients has distinct genetic subtypes that can be treated with available targeted therapies. However, these patients' survival times are shorter than expected due to the more aggressive nature of the disease.
A new study has identified potential genetic alterations in penile cancer, revealing similarities with other squamous cell cancers. The researchers found a common combination of alterations in genes KRAS, HRAS, and NRAS, as well as EGFR, which may impact the tumor's response to an EGFR inhibitor.
A consensus panel of neuropathologists confirms CTE has a distinct pathognomonic signature in the brain, differentiating it from other tauopathies. The criteria define progressive degeneration of brain tissue, including abnormal protein buildup, leading to memory loss and dementia.
Researchers at Houston Methodist Hospital developed a suicide gene therapy that combines radiation treatment with a genetically modified virus to target and destroy cancer cells. The treatment shows high five-year overall survival rates of 97% and 94%, improving upon historical studies by 5-20%.
A new gene therapy platform uses a hydrogel and self-assembled nanoparticles to deliver microRNAs that suppress tumor tissue, increasing dosage at the site while reducing systemic side effects. The approach has shown promising results in shrinking tumors by nearly 90% in pre-clinical models.
A study found that breast cancer survivors with therapy-related leukemia often have inherited cancer susceptibility and gene mutations. Researchers suggest a long-term follow-up study to understand the impact of these genes on leukemia risk, enabling personalized conversations about treatment benefits and risks.
Researchers report improvements in four children treated with gene therapy for Wiskott-Aldrich syndrome, including reduced bleeding events and improved immunologic symptoms. The treatment's long-term safety and efficacy remain to be assessed as the children are monitored for 15 years.
Researchers successfully tested gene therapy combined with low-dose chemotherapy in five patients aged 7-24 with worsening immune systems due to SCID-X1. The therapy showed substantial improvements in immunity and clinical status, particularly in the first two patients who received treatment.
A new gene therapy approach has successfully reduced the number of epileptic seizures in test animals, mimicking human temporal lobe epilepsy. The treatment involves delivering genes for neuropeptide Y and its receptors into the brain, resulting in a significant reduction in seizures for up to 80% of the animals.
Researchers at Stanford University School of Medicine have identified a gene that appears to reduce the risk of heart failure and improve treatment outcomes. The gene codes for a protein involved in regulating sleep, appetite, and blood pressure, which may play a protective role in the heart.
A genetic study of 35,000 patients with inflammatory bowel disease (IBD) reveals new insights into disease progression and severity. The research suggests that IBD may be an array of bowel disorders, rather than a single condition, and could lead to more effective treatments if identified early.
Researchers have made encouraging results using gene therapy to treat cystic fibrosis by inserting a healthy copy of the CFTR gene into affected cells. The approach restored chloride and fluid transport in both mouse models and human-derived cell cultures, suggesting a potential cure for the genetic disorder.
Researchers have identified the Jagged1 gene as a potential target for therapies to combat Duchenne muscular dystrophy, a genetic disorder characterized by progressive muscle degeneration. Increasing expression of JAG1 was shown to prevent the development of degenerative disease in zebrafish models.
Researchers have developed a gene therapy approach that delays symptoms and extends lifespan in dogs with a comparable disease to Batten disease. The treatment involves delivering a working version of a gene to produce a key enzyme, resulting in striking clinical improvements and slowed disease progression.
A study published in JAMA Oncology found that superlatives like 'breakthrough' and 'miracle' were used to describe unapproved cancer drugs, often without clinical data. Targeted therapy was the most commonly referenced class of drugs, with immunologic checkpoint inhibitors also being frequently described as 'game-changers'.
Biotech expert Mitchell Finer shares his experiences and strategies for achieving commercial success, including the importance of clinical data and access to capital. He also discusses the growth of companies like bluebird bio and Cell Genesys, and the potential for gene therapy to revolutionize treatment options.
Researchers at King's College London have identified a new gene, HIFalpha, that regulates nerve signals from damaged mitochondria in fruit flies. Switching off the HIFalpha gene restores nerve function and prevents early failure of nerve cells caused by mitochondrial damage.
A phase III clinical trial's results may lead to the first approved gene therapy product in the US for treating Leber congenital amaurosis type 2 (LCA2). The treatment, SPK-RPE65, could improve patients' ability to see and function at night or in dimmer light levels.
Researchers analyzed genetic material from over 500 CLL patients, identifying dozens of genetic abnormalities linked to the disease. The study found that specific mutations affect treatment response and disease progression, providing insights into the complex interplay between different mutations in cancer cells.
Researchers have developed a new mouse model for spinal muscular atrophy that responds to therapy, allowing for post-symptomatic treatment and potentially improving outcomes for patients. The treatment, an antisense oligonucleotide, restores motor unit function in the muscles even after symptoms have begun.
A study funded by the NIH has shown that gene therapy can preserve vision in dogs with late-stage retinitis pigmentosa. The treatment halted disease-associated cell death and preserved photoreceptor structure, improving visual performance under dim light conditions. Further research is needed to assess its safety and potential toxicity...
A new RNA editing technique called exon skipping has shown promising results in treating a rare and severe form of muscular dystrophy. The treatment, licensed to the Kurt+Peter Foundation, aims to slow down muscle decline and stabilize function in individuals with Limb Girdle Muscular Dystrophy Type 2C.
A Penn study shows that gene therapy can cure canine X-linked retinitis pigmentosa, even when treatment is started at mid- or late-stage disease. The treatment successfully halted photoreceptor cell loss and maintained vision in dogs for over three years.
A University of Copenhagen study reveals that an immune gene, Interferon-beta, may prevent Parkinson's disease and dementia. IFNβ-gene therapy successfully prevented neuronal death in experimental models.
Kazan researchers compared direct gene and blood cell-mediated therapy for spinal cord injuries, revealing similar recovery outcomes in rats. The study found that both therapies preserved myelinated fibers, with cell-based therapy extending therapeutic influence over longer distances.
Researchers develop novel mouse model for Leber hereditary optic neuropathy and demonstrate successful gene therapy in improving visual function. The approach could aid therapy development for various mitochondrial diseases, with a clinical trial currently underway.
Bamosiran reduced IOP by similar magnitude across four doses, but failed to demonstrate non-inferiority to timolol in total population; showed excellent tolerability and non-inferiority at dose 1.125% in patients with baseline IOP >=25mmHg
Researchers characterized two genetic mutations linked to achromatopsia in dogs and found that they destabilized molecular channels essential to light signal transduction. The findings provide new insights into the molecular cause of blindness and present opportunities for conducting preclinical assessments of curative gene therapy.