Articles tagged with Gene Targeting
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A study published in Science Advances identifies a mitochondrial protein called MCJ as the first therapeutic target to preserve cardiac function in pulmonary hypertension. Modulating MCJ levels can activate a signaling pathway essential for adaptation to low oxygen levels, protecting the heart.
A new study reveals that RNA binding motif protein X-linked (RBMX) plays a crucial role in predicting cancer prognosis and response to immunotherapy. Abnormal expression of RBMX is associated with immune regulation, tumor microenvironment, and therapeutic effects of immune checkpoint inhibitors.
A new study found that young adults who reported higher stress as adolescents were more likely to have high blood pressure, obesity, and other cardiometabolic risk factors. This highlights the importance of stress management from an early age to prevent these health issues.
Researchers studied the effects of resveratrol on circadian clock gene expression in young and older human adipose-derived progenitor cells. They found increased levels of some components in older-APCs compared to young-APCs, but also observed gained rhythmicity of some components after resveratrol treatment.
Researchers identified RBM5 as a key regulator of HOXA9 expression in leukemia cells, revealing its dual function in DNA and RNA handling. Removing RBM5 from cells significantly reduced HOXA9 mRNA levels, suggesting its potential as a therapeutic target for acute myeloid leukemia treatment.
A systematic analysis of cancer cells identifies 370 candidate priority drug targets across 27 cancer types. Researchers used machine learning methods to find promising targets and linked them to specific biological markers and genetic features.
Researchers have identified the role of OXR1 in maintaining retromer function, which protects neurons from oxidative damage. Boosting this gene's expression may help extend human lifespan. The study provides potential therapeutic targets to slow brain aging and age-related neurodegenerative diseases.
The study classifies pediatric acute myeloid leukemia (pAML) into 23 distinct molecular categories, revealing unique biological characteristics and drivers of the disease. This classification provides a path forward for clinicians to identify distinct pAML sub-types and guide treatment decisions.
Scientists have created a new generation of prime editors based on the Cas12a protein and circular RNAs, expanding the scope of precision genome editing. The new editors show high editing efficiencies and low off-target effects, paving the way for diverse applications in biological research, disease treatment, and crop breeding.
A Cornell University study using lab mice with human genes found that male mice exposed to arsenic developed insulin resistance and Type 2 diabetes, while female mice did not. The researchers identified a biomarker called miR-34a associated with insulin resistance in Type 2 diabetes.
A recent study published in Aging Cell found a balance between naïve and memory immune cells accelerates or slows down biological aging. The team discovered using novel tools for immune profiling, which opened new doors to understanding the relationships between the immune system and biological age.
Researchers have discovered that natural antimicrobial predatory bacteria, Bdellovibrio bacterivorous, produce fibre-like proteins on their surface to ensnare prey. This breakthrough enables scientists to use these predators to target and kill problematic bacteria in healthcare, food spoilage, and the environment.
Researchers have discovered a brain circuit that mediates panic disorder, consisting of specialized neurons that send and receive the neuropeptide PACAP. Inhibiting PACAP signaling reduces panic symptoms, offering promising findings for future treatments.
Targeted muscle reinnervation performed at the time of amputation reduced pain scores and prevented complications related to abnormal nerve regrowth. The procedure also lowered neuroma risk, particularly in patients undergoing acute TMR.
A new method from Yale University offers a way to analyze how cancer mutations interact with each other, potentially leading to more effective targeted therapies. The approach characterizes the genetic trajectory of individual patients and can help determine appropriate treatments.
Researchers have developed nanodrones that target and eliminate cancer cells by recruiting natural killer cells to tumor sites. The study offers a potential solution for intractable types of cancers, with promising results in suppressing tumor growth without causing side effects.
A drug screening system modeling cancers with lab-grown tissues called organoids helped uncover a promising target for future pancreatic cancer treatments. Researchers identified an existing heart drug, perhexiline maleate, that powerfully suppresses the growth of pancreatic tumor organoids.
A clinical trial found that diosmin reduced swelling at the knee, calf, and thigh up to 14 days after TKA, with lower pain scores on movement. The study also showed improved outcomes for short-term complications and a trend toward decreased postoperative nausea and vomiting.
Mayo Clinic researchers used genetic sequencing to study the measles virus's spread in a human brain. The study found that the virus acquired distinct mutations that drove its spread from the frontal cortex outward. This knowledge may help develop effective antiviral drugs to combat SSPE, a rare and lethal brain disease caused by measles.
Scientists at St. Jude Children's Research Hospital have discovered that only about 80 amino acids in the β2-adrenergic receptor contribute to its pharmacological properties, with specific residues controlling efficacy and potency. Understanding these molecular origins can help design more potent and efficacious drugs.
A new study found that removing the RUNX1 transcription factor and its target gene can lead to a network collapse, causing cancer cell death in a type of aggressive leukemia. This breakthrough identifies specific protein targets for potential treatments.
The study identifies FAM53C as a cytosolic-anchoring inhibitory binding protein of the kinase DYRK1A, regulating its activity and cellular location. This finding may provide potential clinical insights into treating Down syndrome and related diseases.
Scientists at the University of Washington School of Medicine developed a novel protein design approach using AI, creating proteins that bind to challenging biomarkers with exceptionally high affinity and specificity. The breakthrough has implications for drug development, disease diagnosis, and environmental monitoring.
Researchers identified Elovanoid-34, a molecule that modulates the activity of TXNRD1 protein, which regulates antioxidant defenses. This discovery opens new therapeutic avenues for degenerative brain and eye diseases, as well as promoting healthy aging.
Researchers found that pre-zygotic germline variants and post-zygotic hypermethylation at 11p15.5 predispose patients to bilateral Wilms tumor. This study provides new insights into the genetic and epigenetic mechanisms underlying the disease, with potential implications for treatment decisions and clinical trials.
Researchers develop a versatile imaging system for targeted spectroscopy in the eye fundus, allowing for continuous color imaging and spectral measurements. The system enables users to select targets and move them to any location within the eye fundus region without realignment or fixation changes.
Researchers found that blocking cholesterol production prevented progression of serrated-type intestinal tumors in mice, suggesting a potential new strategy for treating these aggressive cancers. The study's findings support the use of cholesterol-lowering drugs to prevent or treat these tumors.
A comprehensive review of targeted therapies for lupus nephritis discusses the challenges of current treatments and proposes strategies to overcome obstacles. Recent advancements in B-cell targeting and alternative approaches such as CAR-T cells are highlighted.
A U of M-led study introduces an innovative genetic engineering method that avoids cost and safety concerns associated with viral vectors. The method combines CRISPR-Cas9 precision genome editing and a novel DNA integration mechanism, integrating large DNA sequences into human T-cells with high efficiency.
Researchers at UVA Health System created an 'atlas of atherosclerosis' revealing critical processes that form harmful plaque buildup. The study provides unprecedented insights into atherosclerosis and its impact on coronary artery disease, heart attacks, and strokes.
Researchers at University of Cincinnati Cancer Center present Phase 2 clinical trial results for a new BTK inhibitor treatment that offers potential for improved efficacy and safety in chronic lymphocytic leukemia. The study also explores the use of IRAK4 inhibitors to target acute myeloid leukemia cells, with promising results.
Researchers leverage AI to analyze healthcare data and identify new targets for effective therapies and accelerate drug development in aging research. AI can tailor cancer treatment more precisely to individual patients' unique aging profiles, optimizing treatment outcomes and minimizing risks.
Researchers at Salk Institute discovered how anti-cancer drugs can prevent fibroblast activation, a protective barrier around pancreatic tumors. The therapy reduces tumor growth and slows disease progression in mice and human patients, offering a promising treatment for pancreatic cancer.
Researchers have created the first extensive map showing which genetic changes can cause disease, leading to valuable insights into neurodevelopmental disorders and cancer. The study reveals that 90% of previously unexplained genetic changes' impact on health is significant, promising speedier diagnosis and new treatment avenues.
Researchers used AI-selected natural images and synthetic images to probe visual processing areas of the brain, finding that predicted maximal activator images significantly activated targeted areas. The study suggests individualized models for each subject can improve understanding of visual system organization across populations.
Research found a significant linear relationship between age of coronary heart disease onset and dementia, highlighting the detrimental influence of premature cardiovascular events on brain health. Adults diagnosed with coronary heart disease at a young age may be at increased risk of developing dementia, Alzheimer's disease, and vascu...
Researchers found an inverse relationship between axon loss and demyelination, suggesting that 'bad' myelin can be more damaging than its absence. This study identifies potential therapeutic targets for diseases associated with myelin defects and inflammation in the nervous system.
Scientists at St. Jude Children's Research Hospital identified genes directly regulated by the oncogenic HOXA9 protein in high-risk pediatric leukemias. The study found two major targets, FLT3 and CDK6, which can be therapeutically targeted with drugs, showing promising outcomes in preclinical models. Additionally, researchers discover...
Researchers discuss CRISPR's limitations in generating accurate cancer models, including variable mutations and indels. Despite these challenges, the technology holds promise for cancer research due to its potential for natural selection and Darwinian evolution.
The American Heart Association and Neurocritical Care Society emphasize the need for improved critical care management of patients after cardiac arrest. The consensus statement highlights gaps in knowledge, particularly in areas like neurological, cardiac, and gastrointestinal care.
Researchers discovered an alternative immune response involving NK and CD4+ T cells that can recognize and attack cancer cells when the usual recognition marker B2M is missing. This finding holds potential for developing more effective combination cancer immunotherapy treatments.
Researchers predict that vampire bats will expand their locations in search of more stable climates, potentially linked to a spillover of rabies. The team's work aims to identify and track the bats by traveling to Colombia to contain the spread.
A team of researchers from Texas A&M University is developing a low-cost, safe, and controlled cancer treatment using programmable bacteria. The $20 million project aims to create an efficient bacterial therapeutic that can target cancer cells with high precision, reducing side effects and costs.
Researchers developed novel small molecule inhibitors of CPSF3, a key regulator of transcription termination in ovarian cancer cells. These inhibitors exhibited potent antiproliferative effects and suppressed tumor growth in vivo.
A team of researchers from Kyoto University found that the pituitary vasopressin system is crucial for building a robust circadian clock in the suprachiasmatic nucleus. This discovery has led to the development of potential treatments for jet lag, which currently target only the suprachiasmatic nucleus.
Researchers have developed a method to camouflage stem cell-derived transplants, avoiding immune rejection and tumor formation. Genetically engineered liver cells can persist in the body despite lack of immune matching, offering a potential solution to organ donor shortage.
A team of scientists has identified the molecular switches involved in halting heart malformations, which could lead to new treatments for tetralogy of Fallot. By targeting the BBLN protein, they aim to prevent defects in four critical areas of the heart.
Researchers found that treating C. elegans with mitochondrial inhibitors extended their lifespan, improved pharyngeal muscle contraction, reduced lipofuscin content, and decreased energy consumption. The study suggests that these drugs could abrogate aging and extend human lifespan, offering a potential therapeutic approach.
Scientists at St. Jude Children's Research Hospital validated GRP78 as a promising but complex target for CAR T-cell immunotherapy. However, they discovered that some tumors trick the immune cells into expressing GRP78, turning off their own cancer-killing ability.
Researchers analyzed genes and brain tissue of patients with Alzheimer's disease to find sex-specific differences in immune function, cellular metabolism, and communication between brain cells. The study suggests that these differences contribute to women's increased risk for the disease and its severity.
A team of researchers at Weill Cornell Medicine used an AI-based approach to uncover patterns among social determinants of health and their impact on children's health outcomes. The analysis found statistical associations between four broad patterns in social determinants and outcomes relating to child developmental health.
Researchers identified a correlation between the HSD3B1 biomarker and resistance to combined hormone therapy and radiotherapy in men with prostate cancer. The study found that high levels of the enzyme led to increased testosterone production, promoting resistance to treatment.
Researchers created a comprehensive chromatin map to understand gene regulatory networks contributing to differences between pediatric acute lymphoblastic leukemia subtypes. The study identified key transcription factor footprints and chromatin accessibility patterns, which can predict leukemia subtype with 89% accuracy.
The American Gastroenterological Association released a new guideline recommending the use of biomarkers to help manage Crohn’s disease. Biomarkers such as C-reactive protein and fecal calprotectin can measure levels of inflammation, helping doctors assess disease activity and guide treatment decisions.
Researchers identified a promising dual-purpose target, KDM1A, using AI analysis of transcriptomic data from 16,740 healthy samples and 11,303 tumors. KDM1A was found to significantly extend lifespan in Caenorhabditis elegans and has anti-cancer activities established in preclinical and clinical studies.
Researchers at Beth Israel Deaconess Medical Center have made a groundbreaking discovery that inhibiting a specific enzyme can halt the progression of Parkinson's disease in a mouse model. The findings suggest that reducing USP30 may slow or prevent PD progression, paving the way for novel therapeutics.
Scientists at St. Jude Children's Research Hospital discovered that a fusion oncoprotein forces cells to become tumor-like, regardless of their original muscle or endothelial cell origin. The study suggests that focusing on the effects of this protein may be a better approach for identifying potential therapeutic interventions.
A new study by Cleveland Clinic researchers reveals significant differences in cell communication between breast tissue with and without BRCA1/2 gene mutations. This discovery provides a foundation for personalized medicine approaches to prevent and treat breast cancer in mutation carriers.
A three-year intensive treatment strategy to lower systolic blood pressure below 120mmHg significantly reduced cardiovascular events, including heart attack, stroke, and death, compared to standard treatment. The study found that participants in the intensive group experienced fewer major vascular events and deaths over a two-year period.
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.