Articles tagged with Gene Therapy
Researchers have developed a groundbreaking gene therapy that targets Diamond-Blackfan anemia, a rare genetic blood disorder. The treatment uses regulated GATA1 expression to correct multiple genetic mutations with a single vector, offering new hope for patients currently reliant on lifelong follow-up care and stem cell transplants.
The CRISPR tool was successfully used to correct a genetic defect in cells affected by chronic granulomatous disease. However, the repair process also introduced new genetic defects, highlighting the need for caution when using CRISPR technology in clinical settings.
The US Department of Defense has awarded $514,000 to study the efficacy of gene editors in treating Duchenne Muscular Dystrophy. Researchers will explore non-viral options for delivering gene therapy through 'self-delivering' gene editors, aiming to improve safety and efficacy.
A new study in mice shows a unique mRNA delivery method can successfully edit faulty genes in fetal brain cells. The technology has the potential to stop progression of genetic-based neurodevelopmental conditions like Angelman syndrome and Rett syndrome before birth.
The ASHG 2024 Annual Meeting will showcase the latest research in human genetics and genomics. The event will feature a Presidential Symposium on Mendelian traits and a Distinguished Speakers Symposium on the promise of human genetics and genomics, among other sessions.
Researchers aim to improve gene therapy design for life-long correction of genetic diseases, but face unknowns including immune response and genomic changes. A five-year NIH award will fund an analysis of genetic and cellular determinants of gene therapy longevity.
Researchers have identified a protein called PERM1 that regulates both energy and heart muscle contraction, offering a new therapeutic approach to systolic heart failure. By addressing the underlying problem of weakened heart muscle, PERM1 may help restore cardiac function and improve patient outcomes.
A large-scale study of children with genetic disorders found that thousands benefited from targeted treatments and support after receiving a genetic diagnosis, with over 20% able to start or adjust therapies. Researchers expect this number to grow as new genetic therapies develop.
A clinical trial of 32 patients with cerebral adrenoleukodystrophy found that six years after treatment, most remained free of major disabilities. However, the study also highlighted safety concerns about blood cancers post-treatment, including myelodysplastic syndrome and acute myeloid leukemia.
Researchers have developed a novel gene therapy approach that targets and breaks down faulty ribonucleic acids in the KCNA2 gene, which is associated with recurring seizures. The therapy has shown promise in reducing excessive neuron activity linked to epilepsy.
Researchers review UBA1 loss of function in VEXAS Syndrome, a hematoinflammatory disorder characterized by severe inflammation, cytopenias, and oncogenicity. They explore therapeutic options, including clone-targeting drugs, to combat this challenging disease.
Two UCF cancer researchers, Alicja Copik and Debbie Altomare, have received $100,000 grants from the Florida Breast Cancer Foundation to develop new treatments for breast cancer. They focus on enhancing natural killer cells to fight cancer and harnessing the body's immune system to create new therapies.
Researchers developed a novel approach to optimizing siRNA-loaded lipid nanoparticles using NMR-based molecular-level characterization. Pre-mixed LNPs exhibit superior gene-silencing effects due to a stacked bilayer structure that enhances gene silencing.
A single-dose gene therapy has been shown to significantly reduce bleeding episodes in adults with hemophilia B, with an average reduction of 71% compared to standard treatment. The therapy, which enables the liver to produce clotting factor IX, has been FDA-approved for use in patients with this genetic disorder.
Researchers discovered that DNA methylation patterns, like cellular memory markers, prevent reprogrammed cells from fully adopting new identities. This limitation limits the effectiveness of long-term treatments and therapies.
A new NIH-funded project aims to address gene therapy's ethical and policy challenges, gathering information from experts in six domains. The study will provide recommendations for addressing these challenges, with implications for patient safety and health outcomes.
Scientists have developed new therapies that selectively remove aggregated tau proteins associated with Alzheimer's disease in mice. The approach utilises TRIM21 to target tau aggregates, leaving healthy tau intact, and demonstrates potential for other brain disorders driven by protein aggregation.
Genethon has developed an innovative gene therapy vector that effectively targets muscle tissue while reducing the risk of liver penetration. The new capsid design uses AI predictive methodology to improve efficacy and safety, paving the way for more effective treatments for neuromuscular diseases.
Researchers at Boston University discovered a new method to harness self-amplifying RNA to create more effective vaccines. The modified saRNA vaccine protected mice from severe COVID-19 disease with a lower dose than current mRNA vaccines. Longer duration of protein expression and reduced inflammation were also observed.
Researchers at Karolinska Institutet successfully used gene therapy to improve vision in 11 out of 12 patients with Bothnia dystrophy, a form of hereditary blindness. The treatment involved injecting a specially designed virus under the retina, which produced normal protein and restored visual function.
Scientists from Trinity College Dublin have developed a gene therapy that protects retinal ganglion cells and improves their function in animal models of glaucoma. The therapy has also been shown to increase oxygen consumption and ATP production in human retinal cells, indicating enhanced cell performance.
Children's Hospital of Philadelphia researchers have reported encouraging evidence of an effective new gene therapy to treat multiple sulfatase deficiency. The ex vivo gene therapy improved sulfatase production and reduced symptoms associated with the disease in preclinical models.
Scientists from Spirovant Sciences describe a novel adeno-associated virus (AAV) gene therapy called SP-101 that has been optimized for efficient human airway cell transduction. After single dose inhaled delivery, the vector showed consistent expression of a functional and regulated shortened human CFTR minigene.
Researchers at Nagoya University have developed a method to chemically alter siRNAs, reducing off-target effects and improving the safety of siRNA drugs for genetic therapy. By modifying the seed region of siRNAs with formamide, they achieved suppression of off-target effects with higher efficiency than existing chemical modifications.
Researchers developed a gene therapy that restored useful vision to most patients with Leber congenital amaurosis type I, a rare inherited blindness. The treatment showed a 10,000-fold improvement in light sensitivity and improved navigation abilities in patients who received the highest dose.
Experts propose a series of scientific principles and experimental approaches to assess potential carcinogenicity of gene therapies. Data transparency is crucial, with publicly accessible data from viral integrations site studies and clinical settings.
A multi-university research team led by University of Virginia engineering professor Gustavo K. Rohde has developed a system that can accurately spot genetic markers of autism in brain images. The system uses generative computer modeling technique called transport-based morphometry, which reveals brain structure patterns that predict v...
Researchers have developed two new methods to produce circular RNAs, which can silence genes and serve as templates for making therapeutic proteins. These circular RNAs display enhanced stability and biological activity in heart muscle cells and neurons.
A recent study found that Chinese patients with ovarian cancer often carry a specific variant of the RAD51D gene, which can promote tumor growth. The variant also makes these patients more sensitive to PARP inhibitors, leading to a favorable prognosis and potential new treatment methods.
Researchers developed a treatment approach using lipid nanoparticles to deliver mRNA therapy for MSUD, extending survival and reducing serum leucine levels. The study also identified a novel AAV variant with desirable biodistribution properties for targeting peripheral organs.
Researchers at the Broad Institute of MIT and Harvard developed a machine-learning approach to design better AAVs for gene therapy. The tool helps engineer capsids with multiple desirable traits, such as targeting specific organs or working in multiple species. About 90% of predicted capsids successfully delivered cargo to human liver ...
The Rice University lab has developed a new noninvasive technique, called REMIS, that can measure gene expression and gene therapy delivery in specific brain regions using ultrasound. This technology could revolutionize brain-based gene therapy for neurodegenerative diseases.
Researchers at the University of Helsinki have created cells that can't grow uncontrolledly, which could lead to new and safer cell therapies for hereditary diseases, myocardial infarction, and other conditions. The innovation uses thymidine supplementation to regulate cell division, eliminating the risk of cancer.
β-Thalassemia patients experience iron overload due to increased intestinal absorption and transfusions, leading to oxidative stress and tissue damage. Chelation therapy is the cornerstone of treatment, but novel therapeutic strategies are needed to optimize patient outcomes.
Researchers found correlations between MIF and DDT levels with patient survival outcomes in melanoma patients. Higher CD74:MIF and CD74:DDT levels were associated with improved survival and enrichment of inflammatory markers.
Researchers at the Mayo Clinic Comprehensive Cancer Center discovered a new treatment approach that improved survival rates for patients with B-cell precursor leukemia by nearly 60%. The study found that adding blinatumomab to chemotherapy reduced the risk of leukemia recurrence and death.
A recent study found that 'gene misbehaviour' is a common phenomenon in the healthy human population, with over half of inactive genes showing misexpression. The researchers used advanced techniques to analyze blood samples from 4,568 healthy individuals and identified mechanisms behind these gene activity errors.
Researchers developed a triple-adeno-associated virus vector system to deliver full-length dystrophin protein into muscles, restoring muscle health and strength in mice models with Duchenne muscular dystrophy. The approach has shown promise for treating patients with the genetic disorder.
A new study published in Nucleic Acid Therapeutics found that siRNA reduces huntingtin mRNA levels in the cytoplasm but not in the nucleus of mouse brains, suggesting a limitation in its effectiveness for treating Huntington's disease. The research highlights the importance of understanding the structure and function of nuclear RNA to ...
Researchers found that subretinal adeno-associated virus 8 injections trigger proinflammatory T-cell responses, but co-injection of immunodominant peptides can modulate the immune system. This study suggests a new approach to AAV gene therapy for retinal diseases.
Researchers at UW Medicine have developed a new gene therapy that delivers protein packets to replace defective genes in muscles, halting disease progression and reversing pathology. The therapy uses adeno-associated viral vectors and aims to restore normal muscle health, with human trials expected to begin in two years.
Researchers describe redundant innate immune pathways triggered by AAV vectors, including sensing of viral genome and cytoplasmic DNA sensors. The study highlights the need to understand complex biologic mechanisms underlying adverse reactions to AAV vectors in human gene therapy trials.
Researchers at Chiba University have discovered a new mechanism of ILC2 immune cell development, which may exacerbate allergic diseases. The study found that the induction of GATA3 expression by an ILC2-specific super-enhancer is essential for ILC2 differentiation.
Researchers at UNC School of Medicine have identified a small molecule that could potentially treat Angelman syndrome by 'turning on' the dormant paternally-inherited UBE3A gene copy. The compound, (S)-PHA533533, has shown excellent uptake in developing brains and bioavailability, making it a promising lead for gene therapy.
A team of Penn State researchers has created a modular genetic circuit that turns cancer cells into a 'Trojan horse,' causing them to self-destruct and kill nearby drug-resistant cancer cells. The circuit outsmarts resistance and eliminates it before the cancer cells can evolve.
A groundbreaking clinical trial has shown that gene therapy can halt the progression of spastic paraplegia type 50 (SPG50) in a young boy. The treatment, delivered via spinal fluid, eliminated serious side effects and showed potential signs of improvement.
Researchers found that inhibiting the AGTR1 receptor with losartan disrupted cancer growth, slowed development and prolonged survival in mice. This targeted gene therapy also protected the heart against chemotherapy-induced cardiotoxicity.
A first-of-its-kind study compared preventative therapy efficacy between two groups: gene therapy alone and in combination with risdiplam or nusinersen. Dual therapy showed promise in independent sitting outcomes, but not in walking age or muscle disease progression prevention.
Researchers at MD Anderson Cancer Center have identified a small molecule compound that restores physiological levels of telomerase reverse transcriptase (TERT), reducing cellular senescence and tissue inflammation. TERT restoration also spurred new neuron formation with improved memory and enhanced neuromuscular function.
Researchers at Francis Crick Institute develop a new genetic therapy that silences mutated NRAS gene in cells with congenital melanocytic naevus syndrome (CMN), potentially reversing debilitating giant moles. The treatment has shown promising results in mice and could be used to reduce cancer risk in affected children and adults.
Researchers developed a therapeutic approach to replace the missing protein in the brain, reducing symptoms of frontotemporal dementia. The approach uses modified viruses to produce progranulin, which is then released into the bloodstream and crosses the blood-brain barrier, rescuing pathologies in mouse models.
A novel gene therapy has restored hearing function in five children with DFNB9 who were treated in both ears, demonstrating additional benefits compared to a previous trial. The treatment also improved speech perception and sound localization abilities, including the ability to appreciate music.
Scientists discover Epstein-Barr virus alters gene regulation in nasopharyngeal cancer cells, leading to rapid tumour growth. The study offers new insights into the link between EBV and NPC, shedding light on intricate processes underlying disease progression.
A team of researchers from Xi'an Jiaotong-Liverpool University has engineered a short sequence of artificial DNA to target the mutant protein p53-R175H, linked to lung, colorectal, and breast cancers. The new molecule, dp53m, inhibits cancer cell growth and increases sensitivity to chemotherapy agent cisplatin.
Researchers at Edith Cowan University have found a significant genetic connection between Type 2 Diabetes and certain gut disorders, which could lead to more effective therapies. The study uncovered shared biological origins with Type 2 Diabetes among gut conditions like stomach ulcers and irritable bowel syndrome.
The study highlights the importance of protease-activated receptors (PARs) in cancer growth and development, with PH-binding motifs identified as a key platform for drug design. The researchers suggest that targeting PARs could provide an alternative to current oncogenic pathways.
Researchers discuss the benefits of CAR-T therapy in treating B-cell lineage acute lymphoblastic leukemia (B-ALL) in children. The therapy, tisagenlecleucel, has shown promising results and is now priced at $508,250, a more manageable cost compared to other gene therapies.
A Japanese research team used machine-learning-driven modular assembly systems to create a more efficient gene editing tool. The study demonstrated an improvement in genome editing efficiency by 5%, showcasing the effectiveness of engineering zinc-finger nucleases through structural modeling.
A new study shows that gene therapy delivered by nanocarriers can repair damaged discs and reduce signs of back pain in mice. The treatment, which uses naturally derived nanocarriers to deliver genetic material for a protein key to tissue development, restored structural integrity and function to degenerated discs.
Researchers have engineered an AAV that efficiently crosses the blood-brain barrier and delivers genes to the brain in humanized mice. This could lead to new treatments for severe genetic brain disorders with no current cures or few treatment options.