Articles tagged with Gene Therapy
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.
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 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.
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 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.
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.
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 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 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 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.
Researchers have identified new mutations in six genes that may explain why some patients respond to a class of anti-cancer drugs known as EGFR inhibitors. These discoveries were made using human tumors grafted into mice, called tumorgraft avatars, which provide a unique environment for studying individual tumor responses to therapy.
Researchers have found five genetic regions that play major roles in cystic fibrosis disease severity, which could lead to new therapies. The study analyzed over 6,000 patients and identified genetic variations associated with lung disease.
A new gene therapy approach delivers the ITGA7 gene to mice with Duchenne muscular dystrophy, reducing symptoms and extending life span by over 10 weeks. The method overcomes previous immunological issues, making it a promising novel treatment for DMD.
Researchers found a promising target for new therapies in genes affected by praziquantel treatment, including a proton pump inhibited by omeprazole. Combining sub-lethal doses of praziquantel with omeprazole killed more parasites than praziquantel alone.
Researchers have developed a novel software that translates the effects of cancer mutations on protein function, revealing how disease mutations target and damage protein signaling networks within human cells. The discovery has significant implications for personalized precision cancer treatments.
Jin Kim Montclare's new research aims to deliver genes and drugs simultaneously, enabling the potential treatment of multi-drug resistant cancer cells. The engineered protein-lipid system breaks down naturally in the body, offering a safer alternative to traditional polymers.
A new enzyme-replacement therapy, Sebelipase Alfa, has been shown to effectively treat Lysosomal Acid Lipase Deficiency by reducing disease-related liver and blood cholesterol abnormalities. The therapy, which targets hepatocytes, resulted in lower cholesterol levels and reduced liver fat content in patients with the condition.
The new 2015 WHO Classification of Lung Tumors provides standard criteria for pathology diagnosis and clinical practice. The updated classification focuses on histologic subtyping and classification according to predominant subtype, leading to multiple new discoveries in lung adenocarcinoma.
Scientists have successfully used gene therapy to fully restore vision in a mouse model of Leber congenital amaurosis-1, a genetic disorder causing severe visual impairment. The treatment, which replaced the deficient retGC1 protein, showed long-lasting results and supports clinical testing for human patients.
A review article highlights barriers to overcome in CAR T cell therapy for lymphoma, including physical barriers and immunosuppression. The study explores factors related to tumor biology and immunology compared to treatment response in patients with lymphoma.
A recent study published in SLEEP reveals that African genetic ancestry is associated with lower slow-wave sleep amounts in African-American adults. The research found a significant genetic basis for racial differences in slow-wave sleep, which may lead to the development of population-specific treatment approaches.
Researchers at Oregon State University have made a significant advance in photodynamic therapy to combat ovarian cancer, achieving complete cancer cell elimination with no regrowth of tumors. The new approach combines existing techniques with compounds that make cancer cells vulnerable to reactive oxygen species, reducing natural defen...
Harvard researchers have reconstructed an ancient virus effective at delivering gene therapies to liver, muscle, and retina. The discovery may lead to novel vectors for safer and more potent gene therapies.
Scientists have developed a new technology that significantly improves the ability to target specific faulty genes and edit them with healthy DNA. The method reduces off-target DNA binding, allowing researchers to produce gene editing tools that are 100 times more specific for their target sequence.
A new test developed by UBC researchers allows physicians to measure the effects of gene silencing therapy in Huntington's disease. The test detects small amounts of toxic protein and can be used to follow changes in brain levels over time.
Researchers reconstructed an ancient virus to deliver gene therapies, targeting liver, muscle, and retina. The discovery could lead to safer and more potent treatments for various diseases.
Researchers have recreated the evolutionary lineage of AAVs to reconstruct an ancient viral particle that is highly effective at delivering gene therapies. The approach could lead to designing a new class of genetic drugs that are safer and more potent than those currently available.
A new study suggests that exogenous glucagon-like peptide 2 (GLP-2) treatment can improve the excretion of toxic bile acids and stimulate liver growth in neonatal pigs with parenteral nutrition-associated liver disease. This finding supports a beneficial role for GLP-2 as a novel therapy in PNALD.
Researchers successfully deliver an RNA encoding alpha-1-antitrypsin (AAT) into cells, enabling them to produce highly functional AAT. This approach offers a safe and cost-effective solution for treating single gene disorders like AAT deficiency.
A modified version of a protein that suppresses female reproductive organ development has been shown to inhibit the growth of chemotherapy-resistant ovarian tumors in animal models. Researchers at Massachusetts General Hospital have developed a gene therapy approach using a viral vector to deliver this protein, which resulted in signif...
Scientists have decoded the genome and transcriptome of an incurable subtype of acute lymphoblastic leukemia, revealing a novel program associated with leukemic cells. The study identifies key genes that trigger a reprogramming of the leukemia cells, leading to promising drug tests, including Venetoclax.
Scientists found that additional genes can compensate for knocked-out genes and mitigate consequences. In a study on zebrafish, researchers identified emilin 3B as a rescuing gene for the egfl7 gene, which regulates blood vessel growth.