Articles tagged with Gene Targeting
The NCI-backed Molecular Targets Platform streamlines and catalyzes drug development by integrating pediatric cancer targets and pathways. This platform empowers researchers, pharmaceutical companies, and advocacy groups to accelerate the pace of drug development for pediatric cancer.
Researchers at Cedars-Sinai Cancer identified genetic signatures linked to patient response to immunotherapy in bladder and other cancers. High DDR1 expression is associated with 'cold' tumors, while high DDR2 is linked to 'hot' tumors.
A new gene therapy approach using the neuroprotective protein SynCav1 has shown promising results in slowing down ALS disease progression and increasing life span in rodent models. The treatment preserved spinal cord motor neurons and extended longevity in mice, with similar effects observed in a rat model of ALS.
Researchers discover gene AVIL responsible for deadly brain tumor also causes two forms of childhood cancer, rhabdomyosarcoma. Blocking AVIL activity prevents formation of the disease in lab samples and mouse models.
Researchers used Guardant NGS to analyze nearly 17,000 lung cancer samples and found MET amplification in 1.2% of cases, with 20.8% having overlapping oncogenic drivers. The study suggests that high gene copy numbers and smaller amplified regions can be used to enrich for the true MET-sensitive population.
Researchers propose a novel paradigm using nanoscale nonlinear fluid dynamics to support recurrent neural networks in neuromorphic computing. The liquid film functions as an optical memory, enabling 'reservoir computing' capable of performing digital and analog tasks.
Researchers have created a new DNA atlas that provides insights into how genes in specific cells contribute to coronary artery disease. The atlas identifies over 200 independent genetic markers associated with disease risk, offering a potential roadmap for interpreting non-coding variants.
A new study published in the Journal of the American Heart Association found that adopting a healthy cardiovascular lifestyle can significantly lower the risk of stroke, even for people at high genetic risk. The study used data from over 11,500 adults and estimated lifetime stroke risk based on polygenic risk scores.
Researchers used deactivated Cas9 proteins to target key segments of the human genome and synthetically trigger gene transcription. The study revealed that enhancers can send messages in both directions, but with a predominant regulatory mode where an enhancer tracks toward corresponding promoters.
A Tel Aviv University study reveals that corals' fluorescence serves as a lure for plankton, which are then consumed by predators like corals. The researchers found that green-fluorescent corals were 25% more preyed upon than yellow-fluorescent ones.
Researchers at Edith Cowan University have found a genetic link between human leukocyte antigens and immunotherapy side effects in non-small cell lung cancer patients. The discovery enables doctors to tailor treatment to individual patients, reducing the risk of toxicities and improving overall outcomes.
A study found that individuals with a rare genetic variant common in people of East Asian descent are at higher risk of vessel reclog during or shortly after endovascular therapy for ischemic stroke. The researchers analyzed data from 277 East Asian adults who received the treatment between 2011 and 2021.
A new study by the University of Pittsburgh found that patients with diabetes on Medicare Advantage plans are more likely to receive preventive treatments, but less likely to receive newer and more expensive medications. This disparity can lead to poorer health outcomes, including higher blood pressure and worse diabetes control.
Researchers have created stem cell models that mimic the genetic disorder, revealing the role of WASP protein in regulating RNA splicing and finding potential therapeutic targets. These findings could lead to new treatments for Wiskott-Aldrich syndrome, a devastating immune deficiency disorder.
Researchers have discovered a unique biochemical profile in severe asthmatic patients, which could lead to more effective treatments. The study found a decrease in carnitine metabolism in severe asthmatics, playing an important role in cellular energy generation and immune responses.
Liu's three-year grant will pursue protein-derived cofactor studies to improve understanding of amino acids and their role in metabolism. The research aims to gain a quicker and more thorough understanding of amino acid function and purpose.
The WVU program trains next generation of toxicologists to collect, analyze air samples from mining, fracking sites, using the university's Inhalation Facility. Students will work with preceptors from various fields to investigate health effects of inhaling toxicants.
Researchers identified a three-gene signature in multiple myeloma tumors that predicts a positive response to selinexor-based therapy. The discovery could improve patient selection for targeted agents and expand the use of the drug into patients who haven't failed other therapies.
A study found that a methionine-deficient diet alters gene expression and DNA methylation in liver cells, increasing the risk of non-alcoholic fatty liver disease. A methionine-supplemented diet had the opposite effect, reducing the risk of liver damage.
Researchers at the University of Cincinnati are studying how lipids help fortify lung cancer cells and if targeting them can lead to better outcomes. The team discovered that lipids play a crucial role in cancer cell growth, membrane fortification, and energy production.
Researchers have identified three distinct brain circuits in the thalamus that contribute to Parkinson's disease symptoms, including motor dysfunction and depression. By manipulating these circuits, they were able to reverse Parkinson's symptoms in mice, suggesting potential new therapeutic targets.
Researchers at Cedars-Sinai have identified a key gene, NLRP11, that plays a crucial role in the innate human immune system. Deleting this gene prevented an immune sensor from triggering an inflammatory response, highlighting its potential as a target for therapies to prevent or treat inflammatory diseases.
A study by Weill Cornell Medicine investigators has revealed the cause and potential precision therapies for a type of aggressive blood cancer called diffuse large B cell lymphoma (DLBCL). The research found that SETD2 gene mutations can lead to an accumulation of DNA damage, causing cancer cells to grow uncontrollably.
New research develops a low-index BaF2 thin film-based microspectrometer technology for LWIR spectral sensing. The study demonstrates the use of flat and stress-free free-standing distributed Bragg reflectors (DBRs) for high-performance wavelength discrimination in the long-wave infrared region.
Researchers developed a genetic test that diagnoses primary immunodeficiency disorders (PID), revealing inherited genetic defects in nearly half of patients. The test uses next-generation sequencing technology to identify specific gene variants associated with PID, enabling targeted treatment and earlier intervention for family members.
A new genome-editing strategy called DAP array can correct dozens of errors at the same time with high precision and efficiency, avoiding off-target edits. The technique leverages tRNA to drive multiple guide RNAs on a single array, then released individually by cells to direct genome editors for edits at multiple human genomic sites.
Researchers at the University of Birmingham have discovered two new DNA repair genes, SETD1A and BOD1L, which can make cancer cells more sensitive to radiotherapy. These findings may lead to improved treatment efficiency and patient outcomes by allowing clinicians to identify targeted treatments for specific patients.
A study estimates that more than 1 million U.S. adults carry a gene for familial hypercholesterolemia, a genetic disorder causing high cholesterol and potentially leading to premature heart attack or death. Genetic testing may help identify those at risk, but its affordability is a concern.
A new screening tool has been developed to identify pregnant women at risk of eating disorders, allowing for early intervention and treatment. The Prenatal Eating Behaviors Screening (PEBS) tool, created by WVU researchers, uses a 12-question screener to assess symptoms and emotions associated with eating disorders.
A new mouse model study found that stress and depression can increase heart disease risk by preventing the beneficial effects of cholesterol-lowering medications. The research suggests that chronic stress can mediate epigenetic changes that make bone marrow precursors more inflammatory, leading to increased plaque formation.
A new study in mice identifies FXR1, a protein implicated in Fragile X syndrome, as a potential target for creating new blood pressure-lowering medicines. Deleting FXR1 in smooth muscle cells resulted in decreased diastolic blood pressure and altered vascular behavior.
Researchers discovered hundreds of new gene functions in algae, which have counterparts in plants, enabling better understanding of photosynthesis, DNA repair, and stress responses. The findings can improve biofuel production and develop heat-tolerant crops.
A recent study reveals that nearly two-thirds of traditional farmer crop varieties are already represented in genebanks, but conservation gaps persist for key crops like pearl millet and potatoes. The analysis highlights areas with high diversity in landraces, such as Bangladesh, Ethiopia, and India.
A gene therapy approach has effectively reduced neuropathic pain in mice with spinal cord or peripheral nerve injuries without detectable side effects. The treatment, which targets impaired neurons, resulted in long-lasting benefits persisting for at least 2.5 months.
A study of historical tiger shark jaws reveals a local southeastern Australian population has been extirpated due to shark control programs, emphasizing the need for regional management and conservation. Genetic diversity drives future evolution, and its loss can lead to reduced adaptability to environmental changes.
A new lab test has been developed by Rutgers scientists to identify COVID-19 variants. The test uses molecular beacon technology and can detect eight different mutations in the spike protein, increasing the transmissibility of the virus and evading immune defenses.
Researchers proposed and experimentally demonstrated an all-optical random bit generation method using chaotic pulses quantized in the optical domain. This method generated a 10 Gb/s random bit stream, potentially operable at higher rates by exploiting ultrafast fiber response.
Researchers at WFIRM have developed a novel method to refine CRISPR/Cas9 gene editing, increasing efficiency and decreasing large DNA deletions. The technique, which fuses DNA polymerase I or the Klenow fragment to Cas9, improves safety and functional editing outcomes.
Researchers have mapped the connections between DNA and blood proteins in two large populations, providing insights into disease causes and potential treatment targets. The study's findings could shed light on health disparities and help develop new therapies.
Researchers at Cedars-Sinai have developed an AI tool that accurately predicts pancreatic cancer patients based on CT scan images. Lowering blood cholesterol has also been shown to slow the growth of prostate cancer by enhancing immune cell action. Additionally, a new study found that an ultrasound probe plugged into a smartphone can b...
Researchers found that genetic mutations in the MAPK pathway, key to normal cell growth, can also make head and neck cancer vulnerable. Individualized genomic analysis can identify specific mutations and target drugs, offering a promising approach to precision medicine.
A new Cambridge study has discovered that the sex of a fetus can affect placenta function, diet-induced maternal obesity, and stress. Designing sex-specific therapies and personalized lifestyle interventions could have lifelong health benefits for children.
The Lustgarten Foundation has awarded a $5 million grant to Break Through Cancer to target KRAS in pancreatic cancer. The Conquering KRAS in Pancreatic Cancer Team will integrate clinical and laboratory approaches to understand why patients respond to new therapies.
Researchers from The University of Texas Health Science Center at San Antonio have identified 33 genes associated with Alzheimer's disease, doubling the known list. This discovery adds 42 new genetic variants to the existing gene list, shedding light on emerging pathways of Alzheimer's biology and potential treatment targets.
Researchers developed a bioinformatics platform called REFLECT to predict optimal combination therapies based on co-occurring tumor alterations. The tool selected combinations that resulted in improved patient outcomes across pre-clinical and clinical studies, leading to increased durable responses.
The University of Cincinnati researcher Xiaoting Zhang has been awarded a five-year grant renewal to continue his genetic driver study of breast cancer and potential therapeutic targets. Zhang's team discovered that the gene MED1 functions as a co-driver with HER2 in promoting tumor growth, spread, and treatment resistance.
Researchers discovered that a genetic mutation causing odd-shaped nuclei may lead to earlier diagnosis and treatment of certain leukemias. The study found that the loss of nuclear Lamin B1 induces defects in nuclear morphology and genome instability, setting the stage for cancer.
The American Association for Cancer Research (AACR) has announced the recipients of its 2022 Scientific Achievement Awards and Lectureships, recognizing Tony Hunter, Christina Curtis, John E. Dick, Kevan M. Shokat, and Lee M. Ellis for their groundbreaking contributions to cancer research.
Researchers have uncovered a collaboration between RNA decay and chromatin regulating complexes that work together to control the levels of transposable element RNAs, preventing genetic instability. The study reveals an unprecedented mechanism of transcriptional and post-transcriptional regulation.
Scientists have developed a new approach to expand the target range of CRISPR/Cas systems, allowing for slight variations in target DNA while maintaining local specificity. This technology could help realize the potential of CRISPR/Cas-based gene therapy and pathogen diagnosis, particularly for diagnostics.
Researchers have created an online resource that maps the cell types and genes expressed in the human and mouse trigeminal ganglion, a key relay center for migraine and facial pain. The atlas identifies potential therapeutic targets that are selectively expressed in cells driving head pain.
Researchers have developed a gene therapy that restores night vision in dogs with congenital stationary night blindness (CSNB), a condition affecting the ON bipolar cells. The treatment enables dogs to navigate mazes in dim light and has a lasting therapeutic effect, paving the way for potential human treatments.
A new study led by Kelly Monaghan at West Virginia University suggests that interrupting the immune response may improve multiple sclerosis outcomes. The researchers found that targeting a specific protein called CCL17 can prevent the disease from attacking the central nervous system, leading to milder symptoms and delayed paralysis.
Researchers have developed a direct method for generating complex structured light through intracavity nonlinear frequency conversion. This technique uses transverse mode locking to produce vortex beams, which are then converted into second-harmonic generation beams with distinct structural characteristics. The study demonstrates the p...
Researchers have described a pioneering chemical technique that can degrade proteins implicated in cancer, potentially increasing the potency and selectivity of new and existing drugs. This technique, known as proteolysis targeting chimeras (PROTACs), targets specific structures within cancerous cells to reduce harmful side effects.
A clinical trial found that genomic sequencing enabled 107 patients to receive matched therapy, increasing treatment options for those experiencing cancer relapse. The study identified previously unknown mutations and showed promise for using circulating tumor DNA to identify targetable alterations.
Researchers at the University of Kansas have identified a new gene target, BUD13, related to Specific Language Impairment (SLI). A family-based study found that rare variants of this gene are associated with SLI in affected individuals. The study suggests that BUD13 may be a key factor in language acquisition and holds potential for ea...
Researchers developed a novel genetic barcode system to mark cancer cells with different gene modifications and image their characteristics. The Perturb-map platform identified specific genes controlling lung tumor growth, immune composition, and response to immunotherapy, offering new approaches for targeting anti-cancer drugs.
Researchers at Cedars-Sinai have identified a potential new therapy for COVID-19, created by reengineered human skin cells. The substance, dubbed ASTEX, stops SARS-CoV-2 from reproducing itself and protects infected cells in human lung cell samples.
Researchers at UCSF develop a new cancer treatment that targets RAS-mutated tumors by exploiting their high levels of ferrous iron. The treatment, TRX-cobimetinib, is more effective and tolerable than current treatments like cobimetinib, which can cause serious side effects in normal tissues.