Articles tagged with Gene Targeting
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Researchers at UCSF describe how to curb MYC levels by disrupting the protein assembly line controlled by RBM42. Disrupting RBM42 in pancreatic cancer cells stopped them from growing, suggesting drugs could be developed to do the same for other fast-growing cancers.
Researchers have developed a new gene therapy that targets aggressive brain cancer, glioblastoma, with a precise delivery system. The treatment uses a novel virus to deliver a targeting drug to cancer cells, achieving cure rates of up to 90% in mouse models.
Researchers have identified a targeted therapy that could bring relief to people living with lichen planus, a chronic inflammatory skin condition. The treatment, baricitinib, selectively blocks specific inflammatory pathways, reducing inflammation and suppressing the overactive immune response that contributes to the disease.
Researchers at King's College London have developed a complex model of molecular 'wear-and-tear' that sheds light on how proteins age. The study found that chromatin, the DNA-protein mix, is more resilient to aging than previously thought, suggesting new avenues for anti-aging treatments.
The American Heart Association teamed up with 20 influential creators to launch American Heart Month and raise awareness about heart health. The event empowered audiences with knowledge about prevention while encouraging donations to support the Association's lifesaving mission.
Researchers have identified ALPK2 as a potential therapeutic target for treating heart failure with preserved ejection function (HFpEF). The enzyme is believed to prevent stiff heart conditions through regulating the TPM1 gene. This discovery offers new hope for developing treatment options targeting ALPK2.
A recent study found that Streptococcus anginosis, a common mouth bacteria, is abundant in the guts of people with acute stroke and linked to a worse prognosis. The researchers also discovered that levels of this bacteria were associated with increased mortality rates two years after stroke.
Researchers at Mayo Clinic developed patient-derived organoid models to study uveal melanoma, a common type of eye cancer. These 3D models accurately represent the disease's genetic and biological characteristics, enabling better understanding and treatment development.
Scientists at Tel Aviv University successfully transport mRNA-based drugs to the immune system of small and large intestines without passing through the liver. The breakthrough could enable treatments for inflammatory diseases such as Crohn's and colitis.
A global team of scientists has made a groundbreaking discovery of a new skeletal tissue called lipocartilage, composed of fat-filled cells that provide super-stable internal support. This unique tissue has immense potential for treating facial defects, birth injuries, and cartilage-related conditions.
The foundation has awarded eight recipients of the 2025 Damon Runyon-Rachleff Innovation Award, including five early-career researchers with initial grants of $400,000 over two years. The awardees aim to develop novel cancer therapies using innovative approaches such as engineered skin bacteria and small molecule-boosted drug delivery.
Researchers used AI to identify genetic factors in Parkinson's disease progression and FDA-approved drugs that can be repurposed for treatment. The study found potential risk genes like SNCA and LRRK2, which are known to cause inflammation, and identified candidate drugs such as simvastatin.
A deadly ciliate parasite has decimated sea urchin populations in the Caribbean, Red Sea, and Indian Ocean, with mortality rates reaching over 90%. The International team of researchers is working to track the disease and preserve sea urchins, which play a crucial role in maintaining coral reef health.
Researchers at Weill Cornell Medicine have identified a specific brain circuit that appears to reduce anxiety without side effects. The study suggests a new target for treating anxiety disorders and demonstrates a general strategy for mapping drug effects on the brain.
Researchers successfully created a bi-paternal mouse by modifying genes involved in reproduction. The mice that reached adulthood exhibited altered growth and shortened lifespan, but could potentially lead to new therapeutic strategies for imprinting-related diseases.
The Au-HN-1 nanosystem offers targeted therapy and dual-mode imaging capabilities for TSCC diagnosis and treatment. By harnessing gold nanoparticles coupled with the HN-1 peptide, this innovative system improves photothermal therapy and enhances tumor detection and surgical margin identification.
The team's novel technique enables high-throughput screening of nanoparticle shapes, sizes, and modifications, reducing associated screening costs. The research demonstrates the distinct preferences of tumour cells for certain nanoparticle configurations, enabling personalized cancer treatments that are safer and more effective.
A new microdosing approach using low doses of buprenorphine has been tested on patients with opioid use disorder, but most found it ineffective. The study, which included 126 participants, showed that only 34% were able to work up to a full dose of buprenorphine.
Researchers have discovered a complex mechanism that allows bacteria to build resistance to antibiotics, involving a KorB-KorA regulatory system. This finding offers a fresh insight into long-range gene silencing in bacteria and provides a potential target for novel therapeutics.
Insilico Medicine has successfully dosed the first patient in its global Phase I clinical trial of ISM6331, a potent pan-TEAD inhibitor. The treatment demonstrates superior efficacy and safety in preclinical studies, with potential synergistic anti-tumor effects and overcoming drug resistance.
UCSF researchers found that when brain cells of female mice express only a maternal X chromosome, their memory and cognitive skills deteriorate faster. This study could explain the variation in brain aging between the sexes and individual women.
Researchers discovered that removing arginase-II gene can slow down muscle aging in mice, leading to improved muscle health and reduced inflammation. This finding suggests targeting the Arg-II gene could help maintain muscle strength and mobility in older adults.
Researchers have identified a key protein called Apex1 as a potential therapeutic target for stopping the immune system from attacking itself. By inhibiting this protein, harmful T cells that cause autoimmune diseases and allergies can be eliminated.
UCSF researchers identify a molecular timer controlling mouse birth timing, which could lead to new tests for human preterm labor risk and interventions. DNA packaging during pregnancy plays a crucial role in regulating gene expression, with KDM6B working as a 'timer' that winds down over time.
A new study demonstrates how fluorescent cholesterol probes can visualize cholesterol in live cells, revealing its role in amyloid plaque formation and cellular signaling. The novel probes have the potential to enhance our understanding of how cholesterol imbalances contribute to neurodegenerative disorders.
Researchers introduced a novel approach to enhance reservoir computing, incorporating a generalized readout that offers improved accuracy and robustness compared to conventional methods. The new method uses a nonlinear combination of reservoir variables to uncover deeper patterns in input data.
A new study published in Aging found that pioglitazone reduced the frequency of skin ulcers in Werner syndrome patients. The research suggests that pioglitazone's anti-inflammatory properties may help wounds heal faster and prevent ulcers from forming.
Researchers at Yale University have developed a new approach that targets multiple pro-tumor actions simultaneously, reducing tumor growth across several types of cancers. The approach uses Cas13 gene editing molecule to silence immune suppression genes and boost antitumor immune responses.
A preclinical study by Weill Cornell Medicine investigators found that an immune protein called the inflammasome helps prevent blood stem cells from becoming malignant by removing certain receptors and blocking cancer gene activity. The study suggests that targeting the earliest stages of cancer may lead to new therapies.
Zebrafish have pineal gland photoreceptors that detect color using parapinopsin 1 (PP1) protein. Two genes, Sagb and Arr3a, play a crucial role in the inactivation of PP1 based on light intensity, with Sagb taking over at higher intensities.
Researchers created new proteins using AI that bind to and neutralize deadly snake toxins, providing a safer alternative to traditional antivenoms. The study's results show an 80-100% survival rate in mice, offering potential benefits for people in developing countries.
A new study explores how city life is influencing the evolution of urban coyotes, revealing genetic changes related to diet, health, thermoregulation, behavior, cognition, and reproduction. The research also highlights the challenges of studying urban coyotes, which are increasingly common in urban areas throughout the US.
Researchers at UVA have developed computer models to target specific bacteria in specific parts of the body, reducing the chance of antibiotic resistance. This approach could lead to more effective treatments and reduce the need for broad-spectrum antibiotics.
Researchers discovered that the protein PIN1 drives bladder cancer by triggering cholesterol synthesis, which fuels out-of-control cell growth. A combination of statins and a PIN1 inhibitor effectively blocks tumor growth in mice, offering a promising therapeutic approach for this deadly disease.
Mayo Clinic is developing foundation models with Microsoft Research and Cerebras Systems to personalize patient care, accelerating diagnostic time and improving accuracy. These multimodal models integrate radiology images and genomic sequencing data to transform clinical diagnosis and treatment.
A recent NUS Medicine study found that a molecule called DUSP6 plays a major role in helping colorectal cancer grow, with higher levels linked to poorer prognosis and decreased survival. Researchers suggest blocking DUSP6 could lead to new therapies for CRC treatment.
Critical Path Institute's Translational Therapeutics Accelerator (TRxA) is launching its 2025 global Request for Proposals to support academic researchers in bridging the 'valley of death' in drug development. The program aims to enable the transition of innovative therapeutics from lab to patients.
Lehigh University bioengineering researcher Tomas Gonzalez-Fernandez is exploring how combining CRISPR with biomaterials can improve gene editing's safety and efficacy for therapeutic use. His NSF CAREER award-funded research aims to develop more targeted and controlled therapies for genetic diseases.
Mayo Clinic's Digital Pathology platform is transforming pathology by leveraging large datasets and AI models for faster, more accurate diagnoses. The partnership with NVIDIA and Aignostics aims to improve patient care and accelerate medical breakthroughs.
Researchers at the University of São Paulo's Medical School have developed a COVID-19 vaccine using virus-like particles (VLPs), which proved highly protective, safe, and versatile in animal trials. The vaccine can be easily updated to combat variants of concern without requiring an adjuvant.
The Menarini Group and Insilico Medicine have entered into an exclusive licensing agreement for a preclinical small molecule targeting solid tumor cancers. The asset has demonstrated broad anti-tumor activity in selected cancers, offering new treatment options for cancer patients with high unmet needs.
Researchers discovered a key role for neutrophils in cancer cell colonization of abdominal fat through the release of DNA webs called NETs. Additionally, a new biomarker study found that dose-dense chemotherapy improved disease-free survival by 20% and overall survival by 15% for women with early-stage ER-positive breast cancer.
A new study found that inhibiting TBK1 increases CAR-T cell activity and makes cancer cells more susceptible to immune cell targeting. The researchers used patient-derived organotypic tumor spheroids (PDOTS) models to investigate the mechanisms of treatment resistance in solid tumors.
Researchers have identified a 177-gene signature common to metastasis across cancers, allowing for personalized risk assessment and potential therapies. The discovery could lead to broader treatment options, faster drug access, and improved patient outcomes.
A multi-institutional study led by Mayo Clinic researchers has improved the accuracy of genetic testing for BRCA2 variants, enabling precise risk assessments and personalized treatment plans. The findings will aid in identifying patients who may benefit from targeted therapies such as PARP inhibitors.
Researchers link neuropilin2 gene to autism and seizure development, highlighting its role in regulating neural circuits. The study suggests targeting specific phases of neuronal development could lead to therapeutic interventions for individuals with autism.
Researchers at Macquarie University have developed a revolutionary new biological pest control method called the Toxic Male Technique (TMT). This approach genetically engineers male insects to produce insect-specific venom proteins in their semen, significantly reducing female lifespan and disease transmission.
A University of Cincinnati Cancer Center study identifies microRNA miR-205 as a promising new target to overcome breast cancer treatment resistance and improve outcomes. The research found that boosting levels of miR-205 could block the production of MED1, a protein responsible for treatment resistance, and improve treatment outcomes.
A recent study found that pregnant individuals with opioid use disorder enrolled in Medicaid are at a disproportionately high risk of severe maternal morbidity. Targeted interventions, such as early Medicaid enrollment and coverage continuity, may be necessary to reduce adverse outcomes in this group.
A study suggests targeting endocan, a protein produced by endothelial cells in blood vessels, could slow tumor growth and make glioblastoma more vulnerable to existing treatments. The discovery may lead to new strategies to combat glioblastoma, which has an average lifespan of just 12-15 months.
Researchers have successfully developed a gene-editing approach using CRISPR-Cas9 to correct the genetic error causing dysferlin protein deficiency, a leading cause of muscular dystrophy. In new mouse models, they restored muscle function and regrowth after transplanting corrected cells.
A recent study has uncovered the crucial role of enzyme G6PC2 in regulating blood sugar levels by controlling glucagon secretion from pancreatic alpha cells. Genetic variations in G6PC2 impact its expression in alpha cells, and inhibiting this enzyme may provide a new therapeutic strategy for type 2 diabetes.
Researchers from Institute of Science Tokyo discovered that tristetraprolin (TTP) regulates inflammatory responses in basophils by promoting mRNA degradation. In TTP-deficient mice, aggravated allergic inflammation was observed, suggesting TTP as a key regulator of basophil-mediated immune responses.
ISM1745, a potentially best-in-class small molecule inhibitor, has been nominated as a preclinical candidate for the treatment of MTAP-deleted cancers. It targets Protein arginine methyltransferase 5 (PRMT5), which is elevated in various cancers and correlates with poor prognostic outcomes.
The ChiTaRS 8.0 database is the world's largest collection of chimeric genes found in humans with cancer and other chronic diseases. It enables the precise adaptation of treatments to specific cancer types, improving treatment success and minimizing side effects.
Researchers at U of T have created SIMPL2, a platform that simplifies detection and improves accuracy of protein-protein interactions. The tool enables the rapid identification of protein interactions, including weak ones, for targeted drug therapies.
City of Hope researchers and collaborators have developed a new method to facilitate encoding numerous features in T cells, allowing for the simultaneous targeting of multiple tumor escape mechanisms. The 'zip-sorting' system doubles genetic information capacity, enabling improved anti-tumor activity and longer-lived T cells.
A new gene editing tool called SPLICER has been applied to reduce the formation of amyloid-beta plaque precursors in a mouse model of Alzheimer's disease. The application shows improved efficiency over current standard gene editing technology and potential for application in other diseases.
Researchers have identified a new pathway for ergosterol biosynthesis in Leishmania parasites, paving the way for more effective antiparasitic drugs. The discovery reveals that azole antifungals targeting a specific enzyme can be highly effective against leishmaniasis.
Researchers at Texas A&M University have developed a skin-like material that can mimic human skin textures and elasticity, simulating conditions for bacterial growth. The Ecoflex-based skin replicas can be used to test wearable sensors and improve catheter designs, potentially reducing the risk of catheter-related bloodstream infections.