Articles tagged with Gene Targeting
Researchers at the University of Washington developed an AI-designed protein that can awaken individual dormant genes by disabling chemical 'off switches'. This approach allows for safe upregulation of specific genes to affect cell activity without permanently changing the genome.
Researchers at Weill Cornell Medicine have identified a protein called HDP1 that plays a critical role in activating genes required for the development of male and female stages of the malaria parasite. Without HDP1, parasites are unable to assemble mature gametocytes, leading to their death and inability to infect mosquitoes.
A new collection of scientific articles reviews the past decade of research on women's cardiovascular health, identifying key differences between men and women. The studies highlight critical gaps in current knowledge and emphasize the need for a more tailored approach to diagnosing and treating heart disease and stroke risk in women.
Codiak BioSciences' exoASO-STAT6 demonstrates potent anti-tumor efficacy by reprogramming tumor-associated macrophages to an M1 phenotype, showing promise as a monotherapy candidate for hepatocellular carcinomas and other cancers. The company plans to initiate Phase 1 clinical trials in the first half of 2022.
CROPSR, an open-source software tool, accelerates CRISPR experiment design and evaluation by addressing challenges in complex crop genomes. The genome-wide approach significantly shortens the time required to design a CRISPR experiment, reducing failed experiments.
Researchers from Trinity College Dublin have pinpointed a key driver gene, SARM1, that contributes to impaired vision and blindness. Deleting this gene shows promise in preserving vision, suggesting targeted therapies may offer long-lasting preservation of sight for various ocular conditions.
Scientists found a protective gene that counters a deleterious mutation causing atrial septal defects, allowing some people with the mutation to thrive. The discovery provides valuable clinical information for families affected by congenital heart disease.
Researchers have discovered a spray-induced gene silencing technique that effectively controls late blight, a devastating disease affecting potatoes and tomatoes. This environmentally friendly method has potential to reduce the usage of chemical pesticides and can be quickly adapted for new targets.
A team of researchers has identified over 250 gene activators in human cells, expanding our understanding of transcriptional regulation and its role in cancer. The study also reveals new insights into how proteins interact with each other to regulate gene expression, potentially leading to the development of targeted therapies.
Researchers at CeMM Research Center discovered that the DNA mismatch repair process plays a crucial role in prime editing. By eliminating mismatch repair, they increased prime editing efficiency by 2-17-fold and improved its accuracy. This fundamental understanding brings the technology closer to clinical applications.
Pulmonary lymphangioleiomyomatosis (LAM) is a rare cancer affecting up to 1 in 1 million women worldwide, characterized by uncontrolled tumor cell growth. Researchers aim to identify new therapeutic targets using extracellular vesicles, with the goal of developing new therapies for LAM patients.
Scientists have developed a gene-silencing tool that can quash gene activity across generations using small noncoding RNA molecules. This technique, called piRNAi, has expanded the molecular toolkit for gene manipulations and allows for more detailed investigations in nematode worms.
Researchers have developed a method to assess drug potential for rare disorders by profiling FDA-approved drugs. The study identified NMD modulators that could potentially treat hundreds of disorders associated with nonsense-mediated RNA decay.
Researchers will use transcriptomics and chemogenetics to identify molecular targets for pain management. The project aims to advance knowledge on pain mechanisms and develop novel therapeutic strategies.
A new study reveals that histone H3.3 plays a crucial role in maintaining the balance between self-renewal and differentiation of blood stem cells, leading to abnormalities when deleted. The protein anchors key epigenetic marks at developmental genes and endogenous retroviruses, contributing to an inflammatory response and skewed produ...
A study led by Clemson University geneticist Allison Hickman has identified 11 high-priority genes associated with uterine cancer. These genes are potential targets for drug therapies, offering new hope for effective treatment options.
Researchers at Uppsala University found that DNA-binding proteins often bind to similar sequences before finding their target, contradicting previous assumptions about gene regulation. This discovery explains how these proteins can rapidly adapt to changing environments without getting stuck on specific sequences.
Scientists have discovered families of proteins that can predict liver transplant rejection, allowing for early detection and modification of immunosuppression. The Blood Proteoform Atlas outlines over 56,000 protein molecules associated with immune cell proteins that change with rejection.
Researchers identified a mechanism by which two antiviral genes, SAMD9 and SAMD9L, promote childhood cancer when mutated. The study found that this protein region performs the toxic function through binding to nucleic acids.
Researchers developed a gene therapy called Targeted Augmentation of Nuclear Gene Output (TANGO), which boosts SCN1A protein production in brain cells. The treatment restored normal cell function and reduced seizures in lab mice with Dravet syndrome, offering hope for the first direct treatment of the fundamental cause.
A new wearable headset, Kernel Flow, monitors brain activity using time-domain fNIRS. The system can record high-resolution brain signals from across the brain with performance similar to conventional systems.
Researchers successfully engineered mesenchymal stromal cells to carry and deliver therapeutics specifically to targeted tissues, offering a precise and reliable approach for treating diseases. This novel cargo-carrier, dubbed 'Cargocytes,' retains most of its cellular functionality while greatly enhancing therapeutic capacity.
Research reveals organic aggregates can emit polychromic and white light with high efficiency, opening up new avenues for OLEDs and encryption. However, more work is needed to fully understand the underlying mechanisms and improve performance.
A team of researchers at George Washington University identified a gene that determines whether ultraviolet iridescence appears in the wings of butterflies. Removing this gene from non-iridescent species leads to UV coloration in their wings, highlighting its critical role in evolutionary differences between species.
Researchers found that even unrelated vaccines can provide cross-protection against multiple pathogens, reducing caseloads and hospital usage. Vaccinating vulnerable populations with other vaccines during the pandemic can have a substantial impact.
The first-in-human trial of CAR-M cell therapy demonstrated that engineered macrophages can target and alter the solid tumor microenvironment, altering the composition of myeloid cells and T-cells. This innovative immunotherapy offers a promising new strategy in the fight against cancer.
Researchers at WVU are studying the Musashi proteins to understand their role in retinal degeneration and develop a universal therapy. By investigating protein translation and gene suppression, they hope to identify potential pathways to boost protein production and slow vision loss.
Researchers developed a non-muscle targeted gene therapy that enhances muscle fiber repair and improves muscle function in LGMD 2B patients. The treatment, administered via a single injection, reduces muscle degeneration and restores myofiber size and muscle strength.
A study from Weill Cornell Medicine and Cornell University reveals that targeting protein ATF4 and its related metabolic protein SIRT3 can trick lymphoma cells into starving themselves, slowing their growth.
Researchers at UC Riverside have discovered that targeting the TNFR1 receptor may be a more effective approach to treating inflammatory bowel disease. By selectively blocking TNFR1, they found significant benefits in mice with Crohn's-like ileitis, suggesting this approach could offer a new opportunity for healing.
Scientists have identified a crucial mechanism for Rhodopsin production in fruit flies, which may lead to a better understanding of retinitis pigmentosa and vision loss. The study reveals that the EMC protein complex is essential for the proper folding and insertion of Xport-A, a key chaperone of Rhodopsin.
Scientists have developed a new method using CRISPR-Cas9 to target specific fat cells, reducing the time and cost of genetic discovery in obesity research. The technique allows researchers to study genes in brown adipose tissue, which plays a crucial role in regulating body temperature.
A study by UC Riverside researchers shows that reactivating the Fmr1 gene in young transgenic mice with Fragile X syndrome eliminates symptoms. This breakthrough treatment offers hope for young children living with FXS and suggests targeting early brain development may be effective.
Researchers at Georgia Institute of Technology have identified a key class of genetic changes associated with cancer, which may be missed by current gene expression analyses. These 'hub genes' play a central role in shaping the network structure of cancer cells and could serve as new targets for targeted gene therapy.
A study found that racial disparities in mammography screening worsened after COVID-19 closures, with non-white patients facing reduced access to facilities. Early interventions aimed at expanding access to these facilities helped to recover late recovery volumes for patients of races other than white.
Researchers at Princeton University have discovered a new compound that can disable the MTDH gene, which is essential for cancer progression and metastasis. The compound shows promise in treating major human cancers, including breast, prostate, lung, and colon cancers.
Researchers developed a novel model to identify specific genes and genetic alterations in multiple myeloma, stratifying the cancer's severity via DNA and RNA sequencing. This model revealed diverse subtypes and high-risk patients beyond current classifications.
Rice scientists developed a comprehensive approach to building better base editors, molecular machines that target and fix faulty DNA at single-base resolution. Their new strategy combines theory and experimentation to pinpoint binding energies and characterize deaminase interactions with ssDNA.
New study suggests inhibiting Shp2 in tumor cells may boost tumor growth and survival, complicating its use as a potential cancer therapy for HCC.
New research enables regionally relevant eating-quality traits to be selected early in breeding programs, saving time and effort. Genetic markers associated with 10 grain-quality traits have been identified, which can now be used by rice breeders in Latin America and potentially worldwide.
Researchers developed a device that uses light to manipulate and isolate individual viruses, enabling precise diagnosis, treatment, and study of viruses. The device has the potential to aid in vaccine development and research on SARS-CoV-2 virus, allowing for targeted analysis of specific virus mutants.
Research suggests that regular PSA testing from age 40 could detect life-threatening prostate cancer in men with genetic hallmarks of Lynch syndrome, increasing the chances of earlier diagnosis and treatment. Men with MSH2 gene faults were eight times more likely to be diagnosed with prostate cancer at a younger age.
A novel computational platform called scAAVengr uses single-cell RNA sequencing to quickly evaluate viral vectors for delivering gene therapies to the retina with maximum efficiency and precision. This approach saves time and resources by identifying suitable candidates that can deliver therapy to affected parts of the retina accurately.
Researchers have discovered three new genetic variants linked to fibromuscular dysplasia, which affects women in their prime and is often associated with high blood pressure and cardiovascular complications. The study provides new insights into the disease's genetic basis and potential therapeutic targets.
The study provides a unique genomic blueprint for understanding the complex mechanisms linking obesity with comorbidities like type 2 diabetes and cardiovascular diseases. The Ossabaw pig's genome is highly relevant to humans, making it an ideal model for studying human obesity.
Researchers have successfully used double-stranded RNA (dsRNA) molecules as a bio-fungicide to suppress the production of a toxin in soybean plants. The study found that dsRNAs produced in bacterial cells can effectively manage fungal diseases, reducing the need for toxic chemicals and potentially mitigating fungicide resistance.
Researchers used CRISPR/dCas9 technology to target DNA methylation and study its effects on cancer cell behavior. The study found that the same epigenetic modification can lead to opposing expression profiles of a target gene in different cancer cell models.
Researchers at Weill Cornell Medicine have profiled individual cells from patients' brain tumors in unprecedented detail, revealing distinct states and programming marks that could be targeted with future drugs. The study offers insights into glioma dynamics and may lead to better detection, staging, monitoring, and treatment methods.
Researchers at Uppsala University have designed new antibodies that bind to both large and small aggregates of the amyloid-beta protein, potentially providing a more effective treatment for Alzheimer's disease. The new antibody format is stronger in binding to clumps and can also target smaller aggregates.
A new study at Ohio State University's Comprehensive Cancer Center is using rapid autopsies to gather biological samples after death to better understand how cancer cells overcome different treatments. This approach has already led to novel findings about drug resistance mechanisms, including the recent approval of a targeted therapy f...
A study analyzed genetic material from 86 discordant couples infected by SARS-CoV-2 and found associations between certain genetic variants and efficient activation of natural killer cells. These cells play a crucial role in the innate immune response, destroying infected cells to prevent disease development.
Researchers at DTU Health Tech have invented a one-pot assay, NISDA, for rapid detection of SARS-CoV-2 RNA without the need for enzyme-based methods. The assay detects low concentrations of RNA in 30 minutes and has shown high accuracy and sensitivity.
Researchers have identified a way to restore the effectiveness of drugs in clinical trials for treating AML by using human alpha(1)-acid glycoprotein (AGP) as a 'decoy' to bind and inhibit FLT3-mutated leukemia cells. The approach has potential for improving patient outcomes, particularly for patients with FLT3-mutated AML.
New targeted therapies are being developed to target genetic alterations in cancer cells, such as the ARID1A mutation found in 10-50% of solid tumours. Early clinical trials suggest that these agents may be effective in treating multiple cancers, including breast, ovarian, and gastric cancer.
Researchers at Johns Hopkins Medicine have identified promising new targets for pancreatic cancer treatment and early detection, including glycosylated proteins that could be captured in the blood for diagnosis. The study also suggests new ways to improve immune response to these tumors.
Researchers found that the gene TCF-1 regulates specific Treg cells, leading to more severe and inflammatory colon cancers. Without TCF-1, these cells become activated and gain a gut-homing feature, resulting in harsher cancer outcomes.
Researchers found that genes can be triggered by specific patterns of light exposure, producing varying levels of activity. The output was not directly correlated to the input, and controlling frequency gave precise control over gene activity.
A CRISPR screening tool identified ZMYND8, an epigenetic regulatory protein, as a potential new therapeutic target for acute myeloid leukemia. Inhibiting ZMYND8 has been shown to leave cancer cells with smaller tumors and better survival in mouse models.
A USF Health study found that deleting a single gene, Foxo1, promotes the growth of functional lymphatic valves in both young and adult mice. This discovery offers a promising early treatment approach for hereditary lymphedema, a chronic condition characterized by fluid accumulation under the skin.
The article considers the ethical issues surrounding enrolling children with neurodevelopmental conditions, such as autism spectrum disorder and fragile X syndrome, in clinical trials. Parents may face difficult decisions about whether to enroll their children due to concerns about potential loss of positive aspects of their condition.