Articles tagged with Gene Therapy
Researchers from the University of Manchester have developed a new stem cell gene therapy to treat Sanfilippo, a fatal genetic brain disease. The treatment has shown promising results in mice, producing near-normal levels of SGSH enzyme and correcting progressive dementia and hyperactivity.
Scientists discovered a molecular switch that regulates regeneration in flatworms, enabling them to grow heads complete with brain, eyes, and wiring. This breakthrough could lead to insights into why some animals regenerate while others don't, potentially informing regenerative therapies for humans.
A team led by Fred Hutchinson Cancer Research Center scientists is developing precision therapies that selectively kill cancer cells while sparing normal tissue. Using high-throughput screening, they aim to identify new genes to target that may be highly specific to each patient's tumor.
Researchers have developed a bioengineered decoy that fools the immune system and prevents it from neutralizing the benefits delivered by a corrective gene. The approach could potentially increase the number of patients who can be treated with gene therapy, offering new hope for genetic diseases like hemophilia.
Researchers have identified key regions in the dengue virus genome that can be targeted to prevent replication. Artificial microRNAs were constructed to specifically target these sites, leading to effective inhibition of viral replication in humans. This innovative approach offers new hope for treating millions affected by dengue fever.
Gene therapy vectors derived from HIV successfully treat metachromatic leukodystrophy and Wiskott-Aldrich syndrome, restoring missing protein and reversing neurodegenerative process. The treatment is safe and effective, offering hope for patients with severe genetic diseases.
A new gene therapy approach using a partially inactivated lentivirus has shown significant improvement in three children with Wiskott-Aldrich Syndrome. The therapy corrects the genetic defect in blood cells by introducing a normal WASP gene, reducing the risk of cancer-promoting genes.
Researchers at Duke University Medical Center developed a blood-based test that accurately identifies who will respond to aspirin therapy and predicts patients at risk for heart attacks. The test, known as the 'aspirin response signature,' measures gene activity patterns in the blood.
A new gene therapy treatment has been developed to cure Sanfilippo Syndrome type A in animal models by introducing the sulfamidase enzyme into cells, eliminating glycosaminoglican accumulation and associated neuroinflammation. The treatment has shown promising results in mice and dogs, with improved behavior and life expectancy.
Researchers have made significant progress in developing new gene therapies to treat Sickle Cell Disease, malaria, and Mucopolysaccharidosis Type IIIA. In the Journal of Clinical Investigation, scientists successfully delivered a replacement gene to the brain in mice and dogs with MPSIIIA using intra-cerebrospinal fluid gene therapy.
At least half of birth-related deafness and many progressive hearing losses have a genetic basis. New sequencing technologies identify 1,000 mutations linked to hearing loss in 64 human genes. This knowledge will lead to practical treatments and effective genetic counseling.
A gene therapy virus has been approved for treating a rare inherited metabolic disease, and researchers found it rarely integrates into the genome, making it safe. The virus was analyzed in patients and mice, revealing its ability to target mitochondria and potentially correcting genetic defects in human mitochondrial DNA.
Researchers develop an engineered virus that can deliver genes to difficult-to-reach cells in the retina, restoring vision to patients with inherited defects and degenerative illnesses. The new therapy is surgically non-invasive and takes only 15 minutes to administer.
Researchers have identified the TTC7A gene as the cause of multiple intestinal atresia (MIA), a rare and life-threatening condition. The discovery will enable the development of prenatal diagnostic tests for pregnant women and screening tests for carriers, offering new hope for affected families.
A new study found that patients with PTSD who have a less functional variant of the BDNF gene respond poorly to exposure therapy. The study suggests that BDNF levels modulate the therapeutic effects of treatments for mood and anxiety disorders.
University of Michigan researchers have discovered how a defective gene in Down syndrome is regulated and its impact on neurological development. By studying fruit fly neurons, they identified two molecular pathways that converge to regulate the gene's abundance, offering a possible therapeutic approach to an aspect of the syndrome.
Researchers at Duke University have developed a novel genetic editing technique that repairs faulty genes responsible for Duchenne muscular dystrophy. The approach, which uses artificial enzymes to modify gene sequences, has shown promise in treating the disease, with over 60% of patients potentially benefiting from this new method.
Scientists use antigen-decorated nanoparticles to prevent immune over-reaction in mice, while also developing a potential gene therapy for Mucopolysaccharidosis Type IIIA. Meanwhile, researchers discover a new target for castration-resistant prostate cancer by blocking mutant androgen receptors.
A subset of metastatic colorectal cancers responds to anti-EGFR drugs but develops resistance within months. The study found that MET gene amplification drives this resistance, and a blood test can detect its presence prior to relapse.
Glybera, first gene therapy drug approved in the Western world, was developed after a decade-long search for its genetic mutation. The European Medicines Agency granted marketing approval on November 2, 2012, after several rejections and appeals. Glybera's efficacy was questionable, but its safety was not an issue.
Researchers have developed a new gene delivery method using magnetically targeted nanoparticles that can effectively deliver genes to injured arteries without causing side effects. The technique, which uses stents as a platform for magnetic guidance, shows promise in overcoming current limitations of gene therapy vectors.
Recent progress toward developing effective gene therapies for regenerative surgery has shown promising results in growing skin, bone, and other tissues. However, translating experimental gene therapy methods into clinical trials remains a difficult challenge due to technical and cost-effective issues.
A study published in JAMA found that a specific genetic variation is associated with improved survival in some cases of pulmonary fibrosis. The researchers identified the MUC5B promoter polymorphism as a key factor, which may lead to the development of new treatments for this deadly lung disease.
The University of Maryland Medical Center is now offering genetic testing to help doctors determine the best medication for each patient, based on their unique genetic makeup. This personalized medicine initiative aims to improve the quality of care provided to cardiac patients.
Researchers at Massachusetts General Hospital have identified a gene variant that predicts weight loss after gastric bypass surgery. The variant, located on chromosome 15, is associated with an average weight loss of nearly 40% in individuals who carry two copies of the beneficial version.
Researchers analyzed 1,647 brain tissue samples to identify molecular networks disrupted in Alzheimer's disease, highlighting the crucial role of TYROBP in immune system dysfunction. This study provides a new framework for understanding Alzheimer's mechanisms and developing potential therapies.
The Hospital for Special Surgery will establish a genomics center to study rheumatoid arthritis and systemic lupus erythematosus using genomic approaches, aiming to develop more effective therapies. The center will focus on epigenetic therapy and personalized medicine, analyzing gene expression and environmental influences.
Researchers have linked 17 genes to juvenile idiopathic arthritis, a significant increase from previous findings of three genes. The study analyzed over 2,800 patient DNA samples and provides new insights into the disease process.
A new treatment for inherited ALS has passed an early clinical trial with no serious side effects, successfully introducing the drug into the central nervous system. The phase 1 trial suggests the approach may be effective against other mutated genes causing central nervous system disorders.
Scientists have discovered 14 new genes associated with childhood arthritis, which could lead to more effective treatments and improved outcomes. The study's findings may also enable clinicians to identify children who require specific therapy earlier, ultimately reducing the impact of the disease on their quality of life.
Researchers from BUSM have developed a genetic signature for COPD from airway cells, providing a new way to study the disease and monitor patient response to treatments. This approach has shown consistent gene-expression changes in both airway and lung tissue cells in individuals with COPD.
A new genetic screen has identified the MKK4 gene as a promising therapeutic target to enhance liver regeneration. The study reveals that inhibiting MKK4 can increase hepatocyte production and survival, leading to healthier livers and improved long-term survival in mice.
A study at Northwestern University and Dartmouth's Geisel School of Medicine found that patients with improved responses to mycophenolate mofetile (MMF) therapy share a distinct gene expression pattern in skin. This signature can potentially guide targeted treatment for patients with scleroderma.
Researchers have developed nanoparticles that improve siRNA delivery, enabling more efficient silencing of specific genes. The approach shows promise in addressing the challenge of targeted delivery for gene therapies.
Using two therapies together has been shown to effectively restore vision in dogs with inherited blindness. The treatment approach may have implications for human patients as well. Researchers believe that combining gene therapy and selective photoreceptor cell destruction could lead to better therapeutic outcomes.
Researchers at the University of Colorado Cancer Center have developed a new technology that uses cholesterol rafts to deliver genetic payloads into cancer cells. This innovative approach overcomes the long-standing challenge of delivering nucleic acids across cell membranes.
A special issue of Translational Research highlights the progress and challenges of gene therapy research, focusing on clinically meaningful studies that combine patient observations with smart experiments. The approach of gene therapy may be applicable to all genetic disorders, offering a promising path for treatment and cure.
Researchers develop a gene therapy technique to increase enkephalin, an opioid compound, in the bladder wall, reducing pain signals. The treatment approach shows promise for severe chronic pain conditions like bladder pain syndrome/interstitial cystitis.
Researchers at Vall d'Hebron Institute of Oncology have successfully eliminated mouse lung tumors by inhibiting the Myc protein, a key regulator of gene expression and cell growth. The study confirms that Myc-targeted therapy is a safe and effective option for treating various types of cancer, with no signs of resistance or side effects.
After over 20 years, a team led by Professor Merlin Crossley has found the genetic puzzle piece for hemophilia B Leyden, a rare disorder that improves symptoms after puberty. The breakthrough demonstrates the importance of persistence and curiosity in scientific research.
A new US patent application allows for methods of restoring visual responses using optogenetic compounds, covering channelrhodopsin and halorhodopsin variants. The approved patent will substantively expand RetroSense's IP estate, providing broad protection for their gene therapies.
Researchers have identified seven new genetic regions associated with AMD, explaining up to 65% of the genetics of the disease. The study combined existing data from over 17,000 patients and 60,000 people without AMD, revealing genes involved in immune system signaling, lipid metabolism, and blood vessel development.
Researchers develop lipid nanoparticles as vectors in gene therapy to deliver therapeutic genes to cells without degrading within the organism. The technology improves drug absorption for insoluble or poorly soluble molecules, offering a promising alternative for diseases with no effective treatment.
A study published in Nature describes how the CPEB1 protein 'takes the brakes off' the production of proteins associated with cell proliferation and tumor progression. The mechanism, discovered using Hodgkin lymphoma cells, has been proposed as a general regulatory system that enhances cancer spread.
A novel drug delivery strategy has been shown to neutralize mutant RNA toxicity and eliminate myotonia symptoms in mice with myotonic dystrophy. The approach uses antisense oligonucleotides and peptide-linked morpholino oligonucleotides to target the disease-causing mutation.
A new gene therapy trial aims to promote the regeneration of heart tissue by encouraging the body to deploy more stem cells to the injury site. The therapy, called JVS-100, has shown clinically significant improvements in exercise levels in patients with ischemic heart failure.
Researchers have discovered a vast potential for noncoding RNAs to be used as therapeutic and diagnostic tools. Long noncoding RNAs play significant roles in various diseases, including cancer, diabetes, and other conditions. The emerging concept of microRNAs functioning as hormones has also led to new avenues for disease treatment.
Scientists have discovered a crucial role of ADAMTS7 enzyme in building up cells in coronary arteries, contributing to the development of coronary heart disease. The study found that a specific gene variant reduces risk by 50% and slows down plaque growth.
Researchers at Case Western Reserve University discovered a missing RNA element that significantly improves the effectiveness of gene therapy. The element, called Genomic RNA Packaging Enhancer element (GRPE), coordinates the production and filling of genetic material in viral vectors.
Aravind Asokan, assistant professor of genetics at UNC School of Medicine, received the award for his significant contributions to gene therapy. His lab developed a synthetic viral toolkit to unravel viral infectious pathways and produce novel vectors for gene therapy.
A landmark clinical trial published in Ophthalmology found that genetic variants do not predict treatment response to Lucentis and Avastin for wet AMD. The study's findings clarify the value of genetic testing for this serious eye disease, contradicting previous hopes for personalized medicine.
Researchers from UAB have shown that gene therapy can cure type 1 diabetes in large animals with a single session. The therapy uses a 'glucose sensor' to regulate blood sugar levels, reducing diabetic hyperglycemia and preventing complications.
Researchers developed a system to artificially simulate natural complex interactions between proteins that regulate genes, allowing for precise control over gene activation. This breakthrough enables scientists to better understand human gene regulation and develop new gene-based therapies.
The EU has announced €38 million funding for research into rare diseases, aiming to develop new diagnostics and treatments through global data sharing. Advances in DNA sequencing have brought personalized treatments closer, but scientists now need to collate data to identify genetic causes of diseases.
A novel aptamer has been developed that specifically targets and stimulates human immune cells, significantly increasing the effectiveness of immunotherapeutic drugs. The aptamer enhances the ability of activated T cells to proliferate and produce immunostimulatory cytokines.
A new study from the University of Pennsylvania School of Medicine found that gene therapy improves vision in patients with Leber congenital amaurosis, but also advances retinal cell degeneration. The research provides a more nuanced understanding of the disease and its treatment.
Researchers have successfully treated Duchenne muscular dystrophy in dogs using gene therapy, reducing inflammation and improving muscle strength. The study, published in Molecular Therapy, provides a quantum leap forward in fighting this devastating disease, paving the way for future human clinical trials.
Researchers developed a new gene therapy approach called exon skipping, which bypasses disease-causing mutations in genes to restore normal expression and protein production. This strategy has shown promising results in treating muscular dystrophy, with improved muscle force and increased body weight and muscle mass.
A new gene has been identified with a key role in obesity and diabetes, regulating fat storage and energy expenditure. The study suggests that blocking this gene could lead to therapies for obesity and related illnesses.
Scientists have identified a rare form of active 'jumping genes' in mammals, specifically in bats. This discovery opens up new avenues for studying evolution and developing tools for gene therapy.