Articles tagged with Cell Therapies
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 phase 1 clinical trial will evaluate the safety and efficacy of CLBR001 + SWI019 in patients with myositis, systemic sclerosis, lupus and rheumatoid arthritis. The therapy has the potential to reduce side effects and patient burden associated with traditional CAR-T approaches.
Researchers developed a dual-action nanotherapy that converts white fat into beige fat and reduces obesity-related inflammation, significantly improving metabolic health without detectable toxicity.
Researchers have identified Tim-3 as a promising therapeutic target for Alzheimer's disease by targeting microglia in the brain. Deleting Tim-3 helps kickstart plaque removal, reduce neuroinflammation, and limit cognitive impairment.
Scientists have discovered a novel regulator of the mitochondrial sodium-calcium exchanger (NCLX), which helps maintain calcium balance in mitochondria. The discovery of TMEM65 could lead to new therapeutic agents to combat calcium overload associated with heart failure and Alzheimer's disease.
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 at Cornell University have discovered key neurological mechanisms in psilocybin's brain effects, targeting pyramidal tract neurons and serotonin receptors. This could enable pharmaceuticals to deliver mood-altering benefits while suppressing perceptual hallucinations.
Researchers found that combining imipridones with radiation therapy and temozolomide slowed glioblastoma growth and prolonged survival in mice. The treatment also boosted immune responses and suppressed MGMT protein expression, making it more effective.
Researchers have discovered a key cellular mechanism regulating mRNA vaccine delivery and stability, proposing a new paradigm for mRNA therapeutics. The study highlights the importance of N1-methylpseudouridine modification in enhancing mRNA vaccine effectiveness.
Researchers found that NSD2 helps maintain MM cell identity by reorganizing DNA and influencing gene activity. This discovery could shape future treatment approaches for patients with t(4;14) myeloma.
A new study suggests that blood cancer patients receiving Bruton Tyrosine Kinase inhibitors should continue their therapy while getting vaccinated against COVID-19. The IMPROVE trial found no improvement in antibody responses when BTKi therapy was paused for three weeks around the time of vaccination.
A team of Chinese researchers identified a novel intercellular signaling mechanism between adipocytes and hepatocytes in endoplasmic reticulum stress response. The study reveals that ceramide, a fat molecule, plays a key role in activating the unfolded protein response pathway in hepatocytes.
A machine-learning algorithm named catGRANULE 2.0 ROBOT identifies molecular targets for further researches and therapies in neurodegenerative diseases. The algorithm analyzes protein-RNA interaction to predict potential harm and identify early pathological signals.
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.
A recent study by the CNIO Melanoma Group has discovered a mechanism by which melanomas evade immune system surveillance. The research found that melanoma cells secrete Midkine, a protein that reduces dendritic cell numbers and reprograms their function to promote tumour development.
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.
Researchers at MD Anderson Cancer Center made several key discoveries, including the spatial organization of cancer-associated fibroblasts across various cancers and a study on treatment resistance in SMARCA4-mutant lung cancer. These findings highlight the importance of investigating cell populations in their spatial context to better...
A team of scientists has created a new method to selectively modify specific proteins in complex biological environments. They achieved this using aptamers and deoxyoxanosine, allowing precise conjugation of desired sites on target proteins. This breakthrough technology has the potential to revolutionize cancer diagnosis and treatment.
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 discovered a potential new treatment for pulmonary fibrosis by targeting the Piezo2 receptor, which plays a critical role in stiffness-mediated profibrotic fibroblast phenotypes. Inhibiting Piezo2 expression or function may slow disease progression and offer new therapeutic options.
Scientists from the University of Zurich created decoy molecules that bind to autoantibodies, preventing them from inhibiting type I interferons. This restoration restores antiviral effect on viruses like influenza, offering hope for treatment and reducing viral disease susceptibility.
Researchers have developed a TIM-3 decoy that improves the effectiveness of CAR-T cell therapy in treating B-cell Acute Lymphoblastic Leukemia. The decoy blocks the interaction between the tumor and immune cells, allowing CAR-T cells to persist and attack cancer cells more effectively.
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.
A new editorial highlights a growing concern in cancer care: radiotherapy may leave behind residual disease that can lead to worse long-term outcomes. Studies have shown that patients with residual disease are more likely to experience cancer recurrence and shorter survival, emphasizing the need for regular biopsy-based tests.
Breast cancer survivors experience accelerated aging due to detrimental effects of BC and its treatments, particularly systemic therapies. The study highlights the need for ongoing monitoring and potential strategies to slow down aging in cancer survivors.
Dr. Nowicki's team has engineered 'supercharged' T cells that produce extra TNF-alpha to boost cancer-fighting ability, offering a potentially more precise and toxic-free treatment option. The new funding will help test these enhanced T cells in preclinical models to evaluate their effectiveness.
Researchers identified PDGFRA as a promising therapeutic target for pediatric high-grade gliomas. Inhibition of the PDGFRA signaling pathway leads to tumor cell death and has shown potential in laboratory and animal models, as well as initial clinical experience with avapritinib therapy.
Researchers have discovered how severe COVID-19 can destroy immune cells' ability to repair the lungs, leading to lingering effects of long COVID. By enhancing damaged organelles using a FDA-approved drug, they found improved lung healing and reduced inflammation.
Researchers found that semisynthetic bile acid NorUDCA inhibits pro-inflammatory T helper 17 cells and promotes anti-inflammatory regulatory T cells in the intestine. This could lead to new therapies for inflammatory bowel diseases such as ulcerative colitis or Crohn's disease.
Researchers at H. Lee Moffitt Cancer Center & Research Institute have discovered a new way to boost cancer immunotherapy by targeting the MARCO protein, which significantly enhances tumor regression in melanoma. This approach could help overcome resistance to current treatments and improve outcomes for patients with treatment-resistant...
Engineered platelets-based nano-aircraft carriers enhance targeted chemotherapy with graded drug release, promoting antitumor immune response and reducing metastasis. Researchers successfully develop Pts-based nanovesicles for precise cancer treatment.
A research team from UH Seidman Cancer Center developed an ultra-fast and highly scalable CAR T-cell therapy manufacturing platform, enabling wider utilization of the therapy. The approach resulted in significantly more favorable toxicity profiles compared to traditional CAR-T products.
A study co-led by the University of Zurich has shown that gluconolactone significantly increases the number and function of regulatory T cells in both mice and humans, promoting a more balanced immune environment. This effect was observed in lupus patients as well, with visible improvements in clinical studies after just two weeks.
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.
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.
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.
Researchers have developed a next-generation CAR-T cell therapy called ALA-CART that can enhance the effectiveness and longevity of cancer cells against harder-to-treat cancers. The treatment has shown promising results in fighting acute lymphocytic leukemias resistant to traditional CAR-T cells.
Researchers discovered that by targeting the Elovl1 enzyme, T cells can harness fatty acid oxidation for energy, increasing their survival and effectiveness against cancer. This metabolic reprogramming approach enhances antitumor activity and improves treatment outcomes in experimental models of melanoma and pancreatic cancer.
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 new way to stimulate cardiomyocyte proliferation, offering promising results in both human cardiac slices and live animals. This innovative approach targets calcium signaling pathways, potentially transforming the treatment landscape for patients suffering from heart failure.
Professor Robert T. Schooley will present a talk at the 8th World Congress on Targeting Phage Therapy, exploring phage therapy advancements and necessary steps for widespread adoption. The congress will gather experts to discuss latest advances, challenges, and clinical applications of bacteriophage therapy.
PARP inhibitors have been found to be effective in treating cancers with BRCA1/2 mutations by blocking DNA repair pathways. The combination of PARPis with chemotherapeutic drugs can also improve treatment efficacy, increasing DNA damage and blocking repair processes.
Researchers have identified a new starting point for therapy by targeting SSAT enzyme in psoriasis, restoring regulatory T cell function and breaking inflammation cycle. This approach could lead to a promising alternative treatment option with fewer side effects.
Researchers discover engineered TIMP molecules can block cancer cell migration and invasion, offering a targeted approach to treating GBM. The findings also suggest the potential for safe treatment with minimal side effects.
A new study reveals that radiotherapy has opposite effects on glioblastoma multiforme (GBM) and low-grade gliomas (LGG), with GBM patients living longer after treatment. The study highlights the need for personalized treatment approaches based on genetic and molecular characteristics to improve survival outcomes.
Scientists have identified a new target to prevent cold sores by understanding how the herpes virus triggers its own immune response. The discovery has important implications for genital herpes caused by the same virus, with potential treatments in development.
Dermatology researchers have identified a novel skin disease in a male patient with erythroderma, a rare and severe inflammation that causes widespread redness and scaling. Targeted treatment with biologic inhibitors reversed symptoms, providing a promising diagnostic tool for precision-medicine.
The ESMO Targeted Anticancer Therapies Congress 2025 features state-of-the-art presentations on new targets, tumour-agnostic drug development, and the role of artificial intelligence in cancer treatment. The congress also highlights recent study results and their potential impact on precision medicine.
A rare case of T cell lymphoma developed in a patient with multiple myeloma nine months after CAR-T cell therapy. Genetic alterations in the patient's haematopoietic cells played a role in tumour development, highlighting the importance of genetic predispositions for potential side effects.
Mutations in MeCP2 gene lead to depletion of NEAT1, a long non-coding RNA controlling autophagy. Restoring NEAT1 reverses cellular alterations in Rett syndrome models.
A new method combines traditional histopathology with spatial transcriptomics data to improve understanding of chronic kidney disease lesions at the cellular and molecular levels. This approach has the potential to identify new biomarkers and therapeutic strategies for patients.
Researchers at UCSF have identified unique, cancer-specific proteins created through mistakes in RNA splicing. These antigens could be used to create potent immunotherapies that recognize and attack hard-to-treat tumors. The discovery offers new hope for glioma patients and expands the number of targets available for cancer therapy.
Researchers at U of T discover a ginger compound called furanodienone that selectively binds to and regulates nuclear receptor PXR, reducing inflammation in the colon. FDN has been shown to increase tight junction proteins, repairing damage to the gut lining caused by inflammation.
Nanomaterials conjugated with drugs improve therapeutic efficacy, reduce toxicity, and enable targeted delivery for neglected tropical diseases. This approach enhances diagnosis with rapid, sensitive, and cost-effective biosensors.
Scientists at Nagoya University have developed a new mRNA technology that can produce up to 200 times more protein than traditional methods. This breakthrough enables the creation of healthy proteins to treat illnesses or toxic proteins to kill unwanted cells.
Researchers at Pusan National University have developed a novel drug delivery system that uses nanoparticles to target and kill colorectal cancer cells. The system, which involves encapsulating cancer cell-activated nanoconjugates in an alginate matrix, can selectively deliver drugs to tumor cells while minimizing side effects.
Researchers at Virginia Tech have developed a method to convert gut bacteria into mini protein factories that produce and release sustained flows of targeted proteins within the lower intestine. This approach eliminates a major roadblock in delivering drugs to this part of the body, offering potential treatment for chronic diseases.
Scientists at NUS Medicine have developed a novel approach using nasal bacteria to deliver therapeutic molecules directly to the brain, reducing appetite and improving glucose metabolism in preclinical studies. The engineered bacteria leverage the olfactory mucosa's unique properties to deliver medication with enhanced bioavailability.
A team of researchers from the University of Arizona College of Medicine – Tucson found that an FDA-approved osteoporosis treatment, risedronate, can correct a gene mutation and normalize heart function in animal models. The study provides hope for treating other rare diseases using precision treatments tailored to individual mutations.