Articles tagged with Gene Therapy
Researchers have identified acid ceramidase as a key enzyme in the production of psychosine, a toxic glycolipid that accumulates in people with Krabbe disease. Removing or inhibiting this enzyme has been shown to decrease or eliminate psychosine production and disease symptoms.
Researchers at Michigan State University have developed a new approach to delivering enzyme-producing genes that can convert certain drugs into toxic agents and target tumors. The study found that nano-bubbles, or extracellular vesicles, were 14 times more effective at delivery and killed over half of the breast cancer cells in mice.
A Nemours study suggests genetic testing can personalize PPI dosing for eosinophilic esophagitis patients. This could improve efficacy and reduce side effects in up to 90% of children, according to researchers.
The team aims to understand how genetics contributes to driving aggressive pathology in prostate cancer by combining pathology, computational modeling, and genomics. They plan to identify therapeutic options by testing combinations of genes in mouse models of the disease.
Researchers have developed a new gene therapy that converts glial cells into neurons, improving motor function in mice and potentially treating stroke. The treatment uses the NeuroD1 gene and has been shown to increase neuronal density and reduce brain tissue loss in mouse models of stroke.
Administering nanoparticles carrying messenger RNA for the arginase gene restored urea cycle function and prolonged lifespan in genetically deficient mice. This treatment approach holds promise for treating inherited metabolic disorders like arginase deficiency.
Researchers used a canine model of Leber congenital amaurosis to study the effects of gene therapy on photoreceptor cells. They found that dogs treated before 63% of their cells were lost had lasting improvements, while those treated later experienced continued degeneration.
Researchers at Ohio State University have made significant breakthroughs in gene replacement therapy for Niemann-Pick type A disease, demonstrating its safety and therapeutic effects in nonhuman primates and mouse models. The treatment has shown a 'bystander effect', preventing motor and memory impairment and increasing survival rates.
Researchers at Cardiff University have identified the CYFIP1 gene as a potential cause of abnormalities in brain structure seen in psychiatric conditions. The study found that the deletion of this gene leads to thinning of the insulation around nerve cells, disrupting communication between brain regions.
Researchers used an optimized AAV9 vector to deliver the galactosylceramidase gene to a mouse model of Krabbe disease, improving clinical symptoms and prolonging median survival. The treated mice lived up to 150 days, compared to 41 days for untreated mice.
A clinical trial found that an oral activator, veledimex, controlled the transcription of human interleukin-12, reducing toxicity while preserving anti-tumor effects. Patients with recurrent glioblastoma had improved overall survival rates, with some experiencing up to 17.8 months of survival.
A new study identifies an enzyme critical for normal wound healing in diabetes, suggesting a potential treatment with allopurinol. In diabetic mice, blocking uric acid production improved healing rates.
Huda Zoghbi, MD, is recognized for her contributions to the field of human genetics, including discoveries of genes responsible for Rett syndrome, spinocerebellar ataxia type 1, and other conditions. Her work has enriched the development of human genetics and its applications in science, medicine, and health.
Scientists at Boston Children's Hospital create human tissue models of CPVT and reverse the condition using gene therapy in a mouse model. The study opens up possibilities for single-dose gene therapy treatments for inherited arrhythmias like atrial fibrillation.
Scientists have discovered that microRNAs play a crucial role in regulating genes in cell nuclei, not just cytoplasm. MicroRNAs targeting the nucleus can increase gene expression, offering a novel approach to gene therapy.
Researchers have identified a gene variant associated with longevity and found it can prevent atherosclerosis by rejuvenating blood vessels. Studies on animal models and human patients showed improved endothelial function and reduced inflammation.
Researchers found that the retina can rebuild itself after treatment, recovering normal light responses and connections to other neurons. Gene therapy successfully restored rod photoreceptors' function in a mouse model with genetically defective rods.
A defective degradation enzyme leads to the accumulation of gangliosides, causing deterioration in patients with storage diseases. Researchers have discovered that genetic disorders can trigger a cascade of consequential damages.
Researchers have successfully developed and tested a gene therapy approach using CRISPR-Cas9 technology to treat Steinert's myotonic dystrophy, a devastating neuromuscular disease. The study showed that the expanded CTG triplet repeat in the DMPK gene was 'cut' and removed from the gene, reducing toxic RNA aggregates in muscle cells.
A recent study by the University of Granada's Tissue Engineering Research Group has found that resident hospital doctors specialising in Family Medicine lack sufficient training on advanced therapies. The researchers also discovered a positive attitude among these doctors towards the use and application of such therapies.
A new roadmap by the Engineering Biology Research Consortium outlines potential breakthroughs in synthetic biology, including genetically modified crops, disease-fighting microbes, and novel biofuels. The report aims to secure federal support for this field to address societal challenges and fuel economic growth.
A team of researchers delivered a therapeutic gene to the spinal canal of infant rhesus monkeys, resulting in sustained expression of the alpha-I-iduronidase enzyme. The study's findings suggest a promising approach for treating severe forms of neuropathic storage diseases.
Scientists at ETH Zurich found that a cellular mechanism called SCF detects and targets alpha-synuclein fibrils for breakdown. This mechanism could be used to develop therapies for neurodegenerative diseases like Parkinson's. Gene therapy and stem cell transplantation may also offer new options.
Researchers have identified a potential therapy for limb girdle muscular dystrophy using lithium to improve muscle size and strength in mice. The findings suggest that inhibiting the protein GSK3beta with lithium chloride can lead to significant improvements in mouse strength and muscle mass.
A new statistical machine learning framework has been developed to reconstruct the original network after a viral attack, which is crucial for designing new medicines or gene therapies against viruses and diseases like cancer. The framework actively incorporates the influence and causality of the attack into its learning algorithm.
The Children's Tumor Foundation has launched the NF2 Accelerator Initiative, a three-year research effort to bring active NF2 treatments to patients. The initiative focuses on expanding the clinical drug pipeline, improving drug selection through innovative testing models, and developing gene therapy options.
Scientists from Sanford Burnham Prebys have revealed a new mechanism for ALS' pathogenesis and suggest that modulating membralin has potential in ALS therapy. A membralin-boosting gene therapy extended the survival of mice with ALS-like symptoms, providing an important new perspective into the disease.
A new clinical trial at IU School of Medicine shows that Burosumab significantly alleviates symptoms of X-linked hypophosphatemia, a phosphate-wasting disease causing rickets and osteomalacia. The study found substantial healing in 72% of children treated with Burosumab versus 6% in the conventional therapy group.
A study of 429 men with metastatic castration-resistant prostate cancer found correlations between specific genetic alterations and clinical outcomes. The loss of the RB1 tumor suppressor gene was significantly correlated with poor survival in patients treated with androgen receptor signaling inhibitors.
Scientists at CU Anschutz Medical Campus identified a rare genetic mutation causing an immune dysregulation syndrome, characterized by recurrent infections and autoimmune complications. The discovery provides unique insights into cell biology and suggests the possibility of treating the disease with gene therapy.
Researchers have developed an algorithm that can predict synthetic lethal interactions between genes, which could lead to more targeted and effective cancer treatments. The Slant algorithm uses data from existing protein networks to identify patterns associated with these interactions.
A new experimental therapy has demonstrated potential in slowing the progression of a genetic form of ALS by reducing SOD1 protein levels. Patients who received the treatment showed improved breathing capacity, muscle strength, and functional abilities compared to those given a placebo.
A researcher at Oregon State University has received a five-year, $3.3 million grant to develop a way for cystic fibrosis patients to get molecular treatment via an inhaler. The treatment would work on every patient, regardless of their genetic mutation, and could vastly improve existing therapeutic methods.
Scientists discovered that sex and diet substantially affect the proteome, a collection of proteins in an animal cell. Understanding these interactions may lead to personalized treatments for humans. The study analyzed large public datasets on human and mouse proteotypes, diet, and genetic status.
A new study compares the effectiveness and cost of gene therapy to allogeneic hematopoietic stem cell transplantation (HSCT) in patients with major beta-thalassemia. Gene therapy was found to be associated with fewer complications and hospital admissions over 2 years, but was nearly twice as costly.
A small clinical trial shows that gene therapy can safely correct the immune systems of infants with X-SCID, a rare life-threatening disorder. The treatment involves inserting a normal copy of the IL2RG gene into the patient's blood-forming stem cells.
Infants with X-linked severe combined immunodeficiency (SCID-X1) have been cured of the disease using gene therapy developed at St. Jude Children's Research Hospital. The treatment produced functional immune cells, including T cells and B cells, in all but one patient.
Researchers found that Hirschsprung disease shares common genetic mechanisms with complex disorders like schizophrenia and autism. The study identified key genes, cellular mechanisms, and molecular targets for personalized treatment.
Researchers at the University of Washington tested the impact of water disinfection methods on antibiotic resistance genes. Three disinfectant methods showed greater than 90% degradation or deactivation, while two others had little to no effect.
Researchers at TGen identified AKR1B10 as a potential biomarker for liver cancer diagnosis and prognosis. The enzyme has a paradoxical role in HCC development, where its overexpression indicates cancer presence while suppression may slow progression.
Researchers at Purdue University discovered a molecule called heparan sulfate that can prevent viruses from escaping cells, raising questions about the safety of gene therapy delivery. The study highlights the need to consider how engineered viruses will exit cells to avoid unintended consequences.
A study found that breast cancer patients who don't respond to targeted therapy have distinct patterns of epigenetic modifications compared to those who do respond. The researchers identified 879 genes with higher methylation levels in resistant patients, suggesting potential targets for new treatments.
Researchers develop new sequencing technologies to identify genetic causes of Opitz C Syndrome, which can cause significant clinical variability. The study's findings suggest that precise molecular diagnosis is crucial for therapeutic intervention and prenatal counseling.
Dr. Roger E. Stevenson, a senior clinical and research geneticist at the Greenwood Genetic Center, has received the 2019 ACMG Foundation David L. Rimoin Lifetime Achievement Award. The award recognizes his vision in founding the center, groundbreaking research on X-linked intellectual disability, and leadership in establishing programs...
Researchers found that some patients' skin areas return to normal as they age, eliminating mutant loricrin genes through somatic recombination. This natural process could lead to a new treatment method for genetic diseases like loricin keratoderma.
Researchers from the CNIO Hereditary Endocrine Cancer Group have identified a new gene, DLST, involved in the development of paragangliomas and phaeochromocytomas. Mutations in this gene were found to be directly linked to the disease, providing a potential breakthrough in diagnosis and treatment.
Researchers have successfully targeted the tumor biomarker EpCAM with chimeric antigen receptor-modified T cells, significantly delaying tumor growth in mice. The study suggests that EpCAM could be a promising target for cancer immunotherapy in various tumor types.
Researchers have identified two new genes, ING3 and EPDR1, that affect bone-forming cells and may lead to stronger bone mineral density and fracture prevention. The study's innovative use of three-dimensional genomic geography analysis could also aid in the discovery of new treatments for other genetic diseases.
Researchers identified a common oncogene, KRAS, as a key player in immune checkpoint blockade therapy resistance in metastatic colorectal cancer. Restoring IRF2 expression or inhibiting MDSCs through CXCL3-CXCR2 signaling increased CRC sensitivity to ICB therapy.
Researchers at UC Berkeley have developed a therapy that can improve and prolong sight in those suffering from vision loss by dampening noise generated by nerve cells in the eye. The treatment has shown promising results in mice with a genetic condition, retinitis pigmentosa.
A multi-center study by MD Anderson Cancer Center reveals that a liquid biopsy test, Guardant360, is comparable to standard tissue biopsies in detecting guideline-recommended biomarkers for advanced NSCLC. The test identified four mutations for which FDA-approved drugs exist and offers faster turn-around time.
A new study found that many commercial cell lines used for laboratory studies are mislabeled as minority ancestry, leading to a lack of diversity and hindering the development of precision medicine for underrepresented populations. Researchers call for more diverse biospecimens to be used in scientific investigations.
The RESTORE Health initiative aims to develop advanced therapies for curative treatments, backed by an international and interdisciplinary community. The consortium plans to invest up to €1 billion in new research projects over the long term.
Researchers identified a genetic mutation in the PKD1L1 gene linked to Biliary atresia, a devastating condition that affects many children. The study found five patients with two copies of mutations and three additional patients with one mutation, providing new insights into the cause of the disease.
The partnership aims to advance genetic diagnosis and therapy for patients with muscle wasting neuromuscular diseases, including motor neurone disease and muscular dystrophy. AIIMS will launch its first patient trials, providing insight into the different genes present in India's populations.
Dr. Carl June is being honored by ACGT for his lifesaving CAR T-cell treatment, which was the first-ever FDA-approved gene therapy for cancer. The funding from ACGT sustained Dr. June's research, allowing it to continue and ultimately lead to promising treatments.
Scientists have successfully restored auditory synapse function and hearing thresholds to a near-normal level in adult mice with DFNB9 deafness. The breakthrough uses an AAV-based gene therapy approach that can be used to produce otoferlin and durably correct the profound deafness phenotype in mice.
A recent study by CU Anschutz researchers has implicated a complex unexamined gene family in autism severity, suggesting that this underexplored area of the human genome may hold key insights into the disorder and potentially lead to new clinical therapies.
Recent advances in understanding Parkinson's disease have identified promising developments to slow or stop its progression. Researchers explore new therapeutic targets, targeting non-motor features of the disease, and repurposing drugs for their potential disease-modifying properties.
Researchers found that silencing microRNA-132 improved insulin secretion and reduced blood glucose in mice and human islet cells. The study suggests antagomir-132 as a potential treatment approach for type 2 diabetes.