Articles tagged with Gene Targeting
STITCHR uses an RNA system to replace entire genes, overcoming CRISPR limitations in targeting every mutation. The tool offers a one-and-done approach for gene therapy, addressing cystic fibrosis and other diseases with thousands of mutations.
A new study by Johns Hopkins Medicine researchers has identified a protein called QRICH1 as a potential target for fine-tuning treatments for cancer and autoimmune diseases. The protein regulates T-cell response, and drugs could be designed to control its activity.
Neuro-immunologist Stephen Hauser has won the 2025 Breakthrough Prize in Life Sciences for his role in identifying the immune system's primary driver of damage to nerve cells in multiple sclerosis. His B-cell theory has led to the development of therapies that have transformed treatment, reducing relapses and improving prognosis.
Researchers investigated why bones become less responsive to exercise with age, finding increased mtROS or decreased Atg7-dependent autophagy in osteoblastic cells do not contribute to reduced mechanoresponsiveness. Damage to the bone's osteocyte network also does not prevent a healthy bone-building response.
Isowalsuranolide activates autophagy-dependent cell death by targeting TrxR1/2, leading to ROS-mediated lysosomal biogenesis and reduced cell growth. The study reveals the TrxR1/2-p53-TFEB/TFE3 axis as a key regulator of cellular homeostasis in cancer.
Researchers at MIT have developed a method to mass manufacture nanoparticles that target cancer cells, eliminating the need for manual polymer mixing and streamlining production. This approach integrates good manufacturing practice (GMP)-compliant processes, making it suitable for large-scale production of cancer treatments.
A new study has revealed that Beagle dogs carrying mutations in the Shank3 gene exhibit face processing abnormalities, similar to those observed in human ASD patients. The research provides direct experimental evidence that mutations in Shank3 lead to ASD-like deficits in face processing, contributing to social impairments.
Researchers at UTA uncovered how the flowerpot snake repairs its DNA and prevents harmful mutations, shedding light on genetic repair mechanisms that could deepen our understanding of human gene evolution. The study also reveals surprising findings about reproductive strategies and immune-related genes in reptiles.
A study explores how large language models (LLMs) can transform teaching by generating customized educational materials, assessing student progress, and providing targeted feedback. LLMs excel at automating routine tasks, reducing teachers' workload and enabling them to focus on mentoring students.
The study highlights ICRAFT's ability to pinpoint genes like TNFAIP3 (A20) with dual immunoregulatory effects in both tumor cells and immune cells.
Researchers discovered senolytics can target Alzheimer's disease-associated brain enzymes AChE and BChE without affecting healthy ones. This selective approach may lead to safer treatments that improve memory and reduce inflammation in older adults.
A multidisciplinary team has generated an atlas to optimize gene therapy delivery, providing researchers with insights into the most effective viral vectors for specific tissues. The study identifies AAV4 as a promising vector for vascular and pancreatic applications, offering new possibilities for treating conditions like diabetes.
The latest APA journal issues explore treatment and risk factors for schizophrenia, as well as medical mistrust and bias. Research also examines the management of borderline personality disorder, including good psychiatric practice and its effectiveness.
Researchers at Cornell University discovered that the FGR protein can induce cell differentiation in leukemia cells similar to retinoic acid treatment. The presence of FGR alone was enough to make these cells mature, producing well-known markers of maturation and expressing inhibitor of the cell cycle p27.
Researchers at Mayo Clinic are conducting a study to understand the factors that contribute to rapid progression of dementia in patients with Alzheimer's disease and Related Dementias. The team aims to identify biomarkers and disease-modifying targets that can improve diagnosis and treatment outcomes for these patients.
Researchers from Prof. Yardena Samuels's lab developed a new approach to cancer treatment by manipulating cancer cells to produce dozens of suspicious proteins, leading to a powerful immune response that destroys human cancer cells and slows tumor growth in mouse models.
The C-Path-led initiative brought together clinical trial sponsors, eCOA technology providers, and regulators to identify and address eCOA implementation issues. Key resources developed during the initiative include guidance on COA data collection and a more open, collaborative environment.
Researchers have identified six promising biomarkers that can detect drug-induced kidney injury earlier than traditional markers, enabling more timely monitoring during drug development. These biomarkers could lead to the development of safer medications and improve patient outcomes.
Researchers discovered TBX2 drives therapy resistance by shifting signaling from the androgen receptor to the glucocorticoid receptor. The study identified a strategy to target this switch, potentially predicting patient risk and offering new treatment approaches.
The Weill Cancer Hub East will unite world-class experts in cancer biology, immunology, nutrition and metabolism to drive pioneering research. The hub aims to improve the effectiveness of immunotherapy, a promising approach for many types of cancers.
Researchers found that digital PCR for BCR::ABL1 is more sensitive and accurate than conventional real-time quantitative PCR to detect ultralow levels of residual leukemic disease. The technology enabled the detection of stable deep molecular remission in 97% of patients, allowing for safe discontinuation of treatment.
Researchers developed a sensor platform that tracks multiple metabolites continuously, offering a window into disease onset and health status. The technology harnesses natural biochemical processes, enabling reliable detection of over 800 metabolites, with potential applications in diagnosing metabolic disorders and optimizing fitness.
A new review highlights how breast cancer cells change their communication and growth patterns, helping tumors survive, spread, and resist treatment. The review explores disrupted signaling pathways, including PI3K/Akt/mTOR and RAS/RAF/MEK/ERK, which can promote tumor progression and resistance to treatment.
Researchers have identified a potential new gene target, FLT1, that could be edited to treat sickle cell disease by increasing fetal hemoglobin levels. The study found 14 new genetic markers associated with fetal hemoglobin production, which could help preserve the type of hemoglobin present at birth.
Researchers mapped m6A patterns across 162 prostate cancer tumors and found that these modifications were closely tied to tumor aggressiveness. Analyzing m6A tags could help doctors predict disease behavior and determine personalized treatment strategies for patients with prostate cancer.
A new study found that recombinant adeno-associated virus (rAAV) capsids contain single-stranded DNA impurities derived from plasmid and host cell DNA. The researchers suggest that the adverse effects of these impurities may differ from those of double-stranded DNA, highlighting the need for further evaluation.
The study of 19,000 patients shows immune checkpoint inhibitors greatly improved survival rates for those living with MSI-H colorectal cancer. Researchers found factors that may improve therapy's effectiveness against MSS tumors under certain conditions.
Researchers at St. Jude Children's Research Hospital found that removing the 'signal jammer' protein VDAC2 can improve how tumors respond to immunotherapy. This breakthrough could lead to new ways to enhance immunotherapies and make them more effective in treating resistant cancers.
Researchers created a powerful new computational and artificial intelligence tool that can generate high-resolution 3D maps of the brain in mice, allowing users to zoom in and out and peer into the full set of molecules producing energy for brain functions. This breakthrough brings scientists closer to understanding the role of metabol...
A team led by Dr. Marcus Botacin is creating a large language model (LLM) to automatically identify malware and write rules to defend against it. The LLM will use signatures to complement human analysts' skills, identifying malware faster and more accurately.
Researchers found that detectable mutant KRAS circulating tumor DNA (ctDNA) indicates a higher risk of cancer spread and worse survival rates for patients with pancreatic ductal adenocarcinoma. The study suggests that ctDNA assays should be performed prior to treatment to have the highest yield.
This study found that aging impacts the gut microbiome composition and metabolites differently in male and female rats, influenced by biological sex and mitochondrial DNA. The researchers analyzed fecal samples from genetically diverse rats to assess bacterial changes with age, revealing more significant shifts in females.
Dr. Owen Witte has been awarded the 12th annual Harrington Prize for Innovation in Medicine for his groundbreaking work on leukemia, lymphoma, and other cancers. His discoveries have led to life-saving therapies like Gleevec and ibrutinib, transforming cancer treatment.
The Chinese Medical Association has released updated guidelines for pediatric mycoplasma pneumoniae infection, emphasizing a multi-faceted diagnostic approach and evidence-based treatment strategies. The guidelines highlight the importance of accurate diagnosis and responsible antibiotic use to combat rising resistance.
Researchers from HKU discover critical molecular changes that drive metastasis to lymph nodes and suppress immune responses, offering promising avenues for new treatment approaches. The study highlights the role of the protein POSTN and TGF-β signaling in tumor progression and immune evasion.
Researchers identified MAFB and CEBPA as critical regulators of urothelial cell growth, highlighting their pivotal role in the genetic mechanisms underlying hypospadias. The study offers new insights into the molecular pathways involved in the condition and paves the way for innovative therapeutic strategies.
Researchers found that suppressing ZNF638 triggers an antiviral immune response in glioblastoma patients, making them more susceptible to treatment. This finding offers a potential new treatment strategy for the disease, which has remained challenging despite recent advances in immunotherapy.
A new bacterial protein, BeeR, has been identified and its structure is being used to develop protein nanoparticles for targeted cancer drug delivery. The protein forms a hollow tube with a cavity capable of containing drug molecules.
Research proposes a new methodology for dynamically optimizing solar panel positioning based on the light needs of crops. Advanced ray tracing techniques are used to evaluate the distribution of solar radiation, enabling the automation of design optimization of agrivoltaic systems.
Researchers have found a way to reactivate the body's natural ability to destroy excess cancer proteins by introducing synthetic RNA fragments that increase poison exon inclusion. This discovery could lead to game-changing therapies for aggressive cancers such as triple-negative breast cancer and certain brain tumors.
A new type of RAS inhibitor, daraxonrasib (RMC-6236), has shown significant promise when combined with immunotherapy in preclinical models of pancreatic cancer. The study found that the treatment not only inhibited tumor growth but also reshaped the tumor microenvironment to make it more receptive to immunotherapy.
Elevated KLF12 levels impair placental development and lead to miscarriages. Researchers propose KLF12 as a biomarker for high-risk pregnancies and explore strategies to normalize its levels.
Researchers have discovered new CRISPR-Cas systems with improved efficiency and specificity, including one from dairy cow bacteria that can target specific gene sequences. The new systems have potential applications in human health, biotechnology and environmental fields.
Researchers found that individuals with lower acylcarnitine levels declined more slowly and showed fewer cognitive decline symptoms compared to those with high acylcarnitine levels. Healthy lifestyle interventions, such as a plant-based diet and exercise, may help lower bioenergetic age and potentially delay or prevent Alzheimer’s onset.
A UMass Amherst Ph.D. student has been awarded a €20,000 grant to investigate a new therapeutic target for Lyme disease by targeting the GuaB enzyme necessary for Borrelia burgdorferi replication in mammals.
Research from the Long Life Family Study found individuals from long-lived families have significantly better vascular health than the general population. The study identified key risk factors and four genomic regions linked to PAD risk, providing novel insight into underlying mechanisms.
A new type of antibody that stimulates the immune system to target cancer cells has shown promise in reducing tumor growth in treatment-resistant breast and ovarian cancers. The study found that the antibody, IgE, uniquely stimulated otherwise inactive immune cells to directly target HER2-expressing cancer cells.
A phase 1 clinical trial led by City of Hope demonstrated the safety and early efficacy of targeted chemotherapy using PIPAC combined with systemic chemotherapy for patients with colorectal and appendiceal cancer that has spread to the peritoneum of the abdominal cavity. The study showed promising results, including histologic, laparos...
The study highlights the role of the Nwd1 gene in liver disease and its potential as a therapeutic target. Mice with Nwd1 gene deletion exhibited liver pathologies mirroring MASH, including excessive lipid accumulation and increased ER stress.
Genetic changes triggered by environmental factors like pollution, diet, and stress can increase cancer risk. Nearly everyone is exposed to cancer risk factors daily, highlighting the need for public awareness and policy action to reduce exposure.
Researchers used CRISPR to cut a single gene from cancer cells of head and neck tumors, resulting in the elimination of 50% of the tumors after 84 days. This groundbreaking study demonstrates that some genes are essential for cancer cell survival, making them excellent targets for CRISPR therapy.
Researchers found that PRP treatment increased gene expression related to metabolism, cell survival, and communication between cells in cumulus cells. This suggests that PRP may help support egg development and fertility in women with poor ovarian response.
A study published in Human Gene Therapy found that over half of individuals with Niemann-Pick disease type C1 lacked neutralizing antibodies against AAV2 and AAV9. This absence of antibodies may impact the effectiveness of gene therapy treatments for this rare disorder.
Researchers have discovered a family of genes essential for tuberculosis survival during airborne transmission, providing new drug targets. The study sheds light on the bacterium's protective mechanisms, which could prevent infection spread and treat existing cases.
A new Northwestern University study found that enhancing the brain's immune cells can clear Alzheimer's plaques and restore a healthier brain environment in immunized patient brains. The findings could reshape the future of Alzheimer's treatments by shifting the focus from removing plaques to harnessing the brain's natural defenses.
Rentosertib, a breakthrough drug candidate for idiopathic pulmonary fibrosis (IPF), has been granted an official generic name by the USAN Council. The drug's development was accelerated using generative AI, identifying TNIK as a promising target and designing small-molecule compounds in just 18 months.
A new brain-mapping technique identified memory-related brain cells vulnerable to protein buildup, a key factor in Alzheimer's disease. The study found that certain cell types in the hippocampus and cortex were more affected by tau buildup.
A new study by researchers at UCSF has found that long-acting injectable treatments can be transformative for patients who struggle to take daily HIV pills, resulting in undetectable viral loads and improved health outcomes. The treatment approach could help stop the spread of HIV by keeping more patients from being infectious.
Researchers discovered that females' dormant X chromosome reactivates in late life, expressing genes related to brain development and cognitive health. This phenomenon may explain why women outlive men and retain their cognitive abilities longer.
This study integrates eQTL, mQTL, and multi-GWAS data to identify potential therapeutic targets of PANoptosis for myocardial infarction. DNA methylation regulates PANoptosis pathways, mediating changes in cardiac diseases.