Articles tagged with Gene Targeting
A single infusion of CRISPR-based gene-editing therapy significantly reduced LDL cholesterol levels in people with heterozygous familial hypercholesterolemia, a condition that increases heart attack risk. The treatment has shown potential as a long-term or permanent option for high LDL-C control.
A randomized controlled trial suggests that a more liberal approach to red blood cell transfusions for heart attack patients with anemia may benefit outcomes without significant risk. The study found no statistically significant difference between two transfusion strategies, but results suggest a possible benefit of liberal transfusions.
A phase 1 trial shows that a single dose of lepodisiran can reduce blood levels of lipoprotein(a) by up to 96%, a key driver of heart disease risk. The therapy, which blocks the production of lipoprotein(a), is well-tolerated and has long-lasting effects.
A new study suggests that a PCSK9 inhibitor given every 1-3 months may effectively lower bad cholesterol levels in people with non-genetic hypercholesterolemia. The medication, recaticimab, showed similar effectiveness and safety as other PCSK9 inhibitors when administered less frequently.
A new therapeutic, lepodisiran, has shown promising results in reducing lipoprotein(a) levels to undetectable levels for nearly one year. The study found that the medication is safe and effective in lowering Lp(a) levels by more than 94%.
A new anti-clotting medication, abelacimab, has significantly reduced bleeding among people with atrial fibrillation who are at risk of stroke. The AZALEA-TIMI 71 trial found that abelacimab substantially reduced bleeding compared to the current standard of care use of direct-acting oral anticoagulants.
Researchers found that people with overweight or obesity who took semaglutide for over 3 years had a 20% lower risk of heart attack, stroke, or death from cardiovascular disease. The study showed significant weight loss and improved cardiovascular outcomes in adults without diabetes.
Researchers at Oak Ridge National Laboratory used quantum biology and artificial intelligence to sharpen the CRISPR Cas9 genome editing tool, improving its efficiency on microbes. The new model revealed key features about nucleotides that enable better guide RNA selection.
A multicenter study identifies genetic and immune-related changes that impact treatment responses in newly diagnosed multiple myeloma patients. The researchers found a complex interplay between tumor genomic features and immune microenvironment changes drives stronger treatment responses and prolonged progression-free survival.
Researchers at Duke University developed a CRISPR-based platform to identify genes that improve T-cell therapies for cancer treatment. They discovered BATF3, a single master regulator of the genome, which reprograms thousands of genes in T cells and greatly enhances cancer cell killing.
The C-Path Neuroscience Annual Workshop brought together stakeholders to chart a transformative course for neurology research and drug development, focusing on chronic progressive diseases such as Alzheimer's and Parkinson's. Key highlights included recommendations for innovative therapies and tools to address complex disorders.
Researchers have identified a crucial biological trigger of Huntington's disease, finding that methylation converts an important protein into waste. By targeting this process, they may develop effective therapies for other neurodegenerative diseases.
A recent study found that transient inflammatory pain causes persistent mitochondrial and metabolic disturbances in sensory neurons, leading to failure in pain resolution. Targeting the cellular redox balance prevents and treats chronic inflammatory pain in rodents.
A recent study has provided significant genomic insight into tar spot of corn, a destructive disease causing $1.2 billion in yield loss. The researchers identified over 100 novel effectors that play a crucial role during infection, warranting further investigation.
A joint research team from South Korea and the US has developed a new genetic classification system for gastric cancer, identifying six consensual genomic subtypes (CGSs) with distinct characteristics. The study suggests potential treatments for each subtype, including immunotherapy and targeted therapies.
A new pathway to prevent cardiovascular disease has been discovered through a University of Minnesota-led study, highlighting the potential of TREM2 as an immunotherapeutic target. The research found that deleting the TREM2 gene in macrophages inhibited atherosclerosis progression.
Mutations in the PIK3CA gene lead to elevated production of the PI3Ka protein, found in approximately 40% of hormone receptor-positive breast cancers. Next-generation PI3Kalpha inhibitors targeting a different region of the mutant protein show promise in overcoming resistance.
Researchers at Weill Cornell Medicine have discovered a new mechanism that makes some cancers treatment-resistant, involving the shuttling of messenger RNAs from the nucleus to the cytoplasm. The approach targets this mechanism with a combination of approved chemotherapies, showing promise in treating persistent cases.
Scientists have created a method to keep targeted particles cool, allowing safe trapping of living cells in their native fluids. This advancement could help overcome problems with current laser light tweezers and enable targeted drug delivery applications.
Researchers developed a new class of drugs targeting PRMT5 enzyme, exploiting genetic vulnerability in tumor suppressor genes. Early clinical trials show promising results for patients with specific types of cancer, including melanoma and mesothelioma.
A new versatile platform controls peptide hormones in fish, revealing surprising findings about conserved genetic networks and adaptable hormone regulation. This innovative method holds promise for transforming fish farming practices and exploring ways to prolong vertebrate lifespan.
A collaborative study has identified key epigenetic targets for treating hepatoblastoma, the most common childhood liver cancer. The research found that the enzyme histone-lysine methyltransferase G9a plays a crucial role in epigenetic regulation and is a promising therapeutic target.
A study led by UC San Francisco found that homeless individuals have a 7 times higher rate of sudden cardiac death compared to the general population. Non-cardiac causes, such as overdoses and unrecognized infections, are more common among homeless people.
A study of 5,435 adolescents and young adults with cancer found that those receiving specialty palliative care experienced a decrease in pain severity. However, the intervention did not affect other symptoms, highlighting the need for new interventions targeting other symptoms during treatment.
Researchers have developed an integrated THz vortex beam emitter to detect rotating targets with remarkable precision. The system uses spiraling electromagnetic waves with orbital angular momentum to accurately measure the speed of a rotating object, with a maximum margin of error of just around 2 percent.
Researchers from Columbia University have validated GLS2's ability to promote ferroptosis in murine models. This study suggests that targeting GLS2 may be a potential therapeutic strategy against liver diseases, particularly hepatocellular carcinoma.
Researchers are testing a single target to weaken tumors and strengthen immune cells in pediatric brain tumor patients. The goal is to improve the effectiveness of CAR-T therapy, which has shown promise but also leads to exhaustion of immune cells.
Researchers discovered that lactate-producing intratumoral bacteria drives resistance to radiation therapy. Cancer cells respond to radiation by rewiring metabolic signaling pathways using lactic acid instead of glucose.
Scientists have developed a technique to restore the function of human-derived GPCR proteins in yeast cells, which could accelerate research and lead to more effective treatments. The approach, using error-prone polymerase chain reaction, introduces random mutations that enhance protein stability and function.
Researchers have assembled the entire genetic code of the brushtail possum, revealing population diversity, reproduction, and origins. The study's findings provide valuable knowledge for managing the animal's populations in both New Zealand and Australia.
Researchers found that deep neural networks often respond the same way to images with no resemblance to the target, generating unnatural signals. The models develop unique invariances that are different from human perceptual systems, causing them to perceive pairs of stimuli as similar despite their differences.
Researchers discovered that targeting TUG1 can control brain tumor growth in mice, suggesting a potential strategy to combat aggressive brain tumors. By inhibiting TUG1, the therapy significantly suppressed tumor growth and improved survival rates when combined with standard treatment.
Researchers from the University of Virginia Health System have made significant discoveries about the genetic influences on fatty acid metabolism in diverse populations. The study found broad similarities among groups but also notable differences, highlighting the need for genetic studies in diverse groups.
Researchers discovered that inhibiting the BRAF/EGFR/MEK pathway significantly reduced cancer stemness and drug resistance in primary colorectal cancer cells. The study also identified a triple combination treatment against BRAF, EGFR, and MEK as a promising approach to block these activities and develop effective treatments.
Researchers at the University of Virginia Health System are developing computer models to better understand the cellular processes and gene activity of multi-drug resistant bacteria Staphylococcus aureus and Pseudomonas aeruginosa. The goal is to identify vulnerabilities in these bacteria and advance the development of new treatments.
A study of over 850,000 Australian children born between 2009 and 2017 found a small increased risk of birth defects among those conceived through fertility treatment, partly attributed to parental infertility. The use of intracytoplasmic sperm injection (ICSI) was also associated with an increased risk for genitourinary abnormalities.
A new American Heart Association advisory introduces the concept of cardiovascular-kidney-metabolic (CKM) syndrome, which affects nearly every major organ. The approach highlights the importance of screening for CKM-related conditions and addressing social factors to prevent progression.
A new national ALS research consortium, Access for All in ALS, has been launched with a $16.7 million NIH award. The consortium will include 34 clinical sites nationwide and generate a longitudinal biorepository linked to detailed clinical information.
Researchers at Scripps Research have identified promising cancer drug targets by combining precise genome engineering and protein activity profiling. They used base editing to alter thousands of possible drug targets, then integrated the data with chemical proteomic information to pinpoint hundreds of potential targets.
A new study examines cannabis use patterns, motivations, and risk factors among college students at a large public university in the southeastern US. The researchers found that top reasons for cannabis use were relaxation and social pressure, while daily users reported higher quantity of cannabis per day.
A coordinated network of space lasers could prevent collisions with manned and unmanned space assets by nudging debris off potential collision courses. The artificial intelligence-powered lasers can maneuver and work together to respond rapidly to debris of any size.
Researchers developed a calculation to identify moderate aortic stenosis patients at higher risk of mortality. The study found that low mean arterial pressure is associated with higher mortality in all patient subgroups.
Researchers developed a novel approach to predict therapeutic targets for aging and age-related diseases. They trained a domain-specific BioGPT model on biomedical literature, which improved its performance in identifying prospective targets.
A team of researchers from Mass General Brigham has developed a monoclonal antibody that reduces abnormal tau proteins associated with Alzheimer's disease in a preclinical model. The therapy, inspired by a genetic variant that provides resistance against the disease, offers an alternative approach to existing treatments.
Research reveals that female fruit flies pre-select for males with superior genes before influencing sperm storage to ensure compatible fertilization. The study provides new insights into the mechanisms and consequences of mate choice, shedding light on genetic variation and species evolution.
A study published in The Lancet Oncology reveals a genetic explanation for increased second-cancer risk and mortality among childhood cancer survivors. The research identified specific genes associated with this increased risk, enabling personalized cancer screening and prevention strategies.
A new drug called vorasidenib has been shown to significantly slow tumor growth and extend the average time until tumor growth in patients with grade 2 IDH-mutant gliomas. This breakthrough could offer a first early treatment option for these cancer patients, potentially improving their quality of life.
A new study by St. Jude Children's Research Hospital predicts the formation of condensates in cancer-associated fusion oncoproteins, which promotes cancer development. The researchers used machine learning to analyze biomolecular features and established a framework for understanding the mechanisms underlying oncogenic properties.
A new strategy optimizes optical and electrical characteristics of thin c-Si solar cells, improving conversion efficiency by 28% compared to industrial thick counterparts. The proposed design uses a layer transfer method and metal nanofilms for enhanced light absorption and surface passivation.
Researchers use Listeria bacteria to break open cell organelles and study their function in relation to shape. They found that function does not always follow form, with cells recognizing and increasing efforts to remove misshapen organelles.
Researchers discovered protein p53's role in regulating sociability, repetitive behavior, and hippocampus-related learning and memory in mice. Lowering p53 levels led to changes in gene expressions related to behavior, while elevated p53 levels were linked to positive learning outcomes.
Researchers found that etripamil nasal spray restored normal heart rhythms within 30 minutes in 54% of patients, making it a potential new option for self-treating paroxysmal supraventricular tachycardia. The study showed the medication to be safe and well-tolerated.
Researchers at Rice University have developed a non-invasive gene delivery technique using ultrasound to efficiently deliver clinically used gene therapy vectors throughout the brain. The study, published in Gene Therapy, shows that opening more sites within targeted regions improves gene delivery efficiency.
A small molecule drug improved the fitness of hematopoietic stem cells used in cell transplants, potentially enhancing the success of procedures like ex vivo gene therapy. The study found that targeting extracellular vesicles relieved stress on cells outside the body, improving their performance when transplanted back in.
Researchers identified potential therapeutic targets for Alzheimer's disease and other conditions using a new approach combining AI-driven target identification with protein phase separation analysis. The study provides insights into the role of protein phase separation in human disease and its potential as a therapeutic target.
Researchers at Mayo Clinic discovered that senescent macrophages in the lung promote tumor growth by blocking the immune system's response to abnormal cell growth. Eliminating these senescent cells delays tumor formation, suggesting a potential therapeutic target for cancer treatment.
A study using engineered mice found that BA.5, a contagious omicron subvariant, replicates rapidly early during infection, causing significant weight loss and high levels of inflammatory cells and cytokines in lungs.
Researchers explore kinase inhibitors as targeted therapies for specific CRC subsets, offering hope for improved treatment options. Key findings suggest that uncovering essential kinases for tumor growth can lead to more effective treatment strategies in metastatic or later-stage CRC patients.
Scientists have identified thousands of non-coding genetic variants linked to Alzheimer's disease, focusing on their impact on microglial gene expression and function. The study found that turning off specific regions can affect multiple genes, highlighting the complex mechanisms underlying AD.
Researchers create adenine base editor with 'on/off' switch, reducing off-target edits by over 70% and increasing accuracy of on-target edits. The tool has potential to correct nearly half of disease-causing point mutations in human genome.