Diseases
Diagnosed at just 8 months old, Elly Krueger's form of Neurodevelopmental Disorder with Regression, Abnormal Movements, Loss of Speech, and Seizures (NEDAMSS) was rapidly progressing, with no known cures or treatments available. Leveraging their expertise and network, the Kruegers formed Elly's Team, a foundation dedicated to
Genetic research
Researchers from the Faculty of Science have made a breakthrough in cancer gene therapy, identifying 10 potential therapeutic candidates for liver cancer. According to the study, liver cancer is a significant health concern worldwide, particularly in developing countries, where it poses a substantial financial burden. The research aimed to uncover
Genetic research
Research conducted at Mustansiriyah University in Iraq has shed light on the role of nanoparticles in enhancing secondary metabolite production in plants. The study focused on the phenylalanine ammonium lyase (PAL) gene family, which is essential for secondary metabolite production and plant defense mechanisms. By analyzing the expression of PAL
Genetic research
Research conducted at the Affiliated Hospital of Qingdao University in Shandong, People's Republic of China, has highlighted the complex role of transforming growth factor-beta (TGF-β) in cancer and fibrosis. The study reveals that TGF-β has opposing effects on fibrosis and cancer at different stages of these diseases. Under
Genetic research
Investigators at Icahn School of Medicine at Mount Sinai have made significant breakthroughs in cancer gene therapy by designing a novel artificial 5' untranslated region (5'UTR) that enhances modified mRNA translation efficiency in cardiac tissue. According to the research, the artificial 5'UTR, termed "Top
Diseases
Andelyn Biosciences, a leading Contract Development and Manufacturing Organization (CDMO), has successfully manufactured a novel viral vector gene therapy using its AAV Curator Platform to treat a baby with a rare neurodegenerative disease. The therapy was produced and released just 10 months from the start of development, a significant achievement
Diseases
Matrix metalloproteinases (MMPs) play a crucial role in extracellular matrix remodeling and are implicated in the pathogenesis of various cardiovascular diseases, including atherosclerosis, myocardial infarction, and heart failure. Recent research highlights MMPs' involvement beyond ECM degradation, influencing lipoprotein metabolism, inflammatory signaling, and intracellular processes critical for cardiovascular homeostasis. Despite
Genes
Researchers at University College London (UCL) have reported significant advancements in the field of gene editing, citing improved efficiency and potential for curative genetic medicines. A key development in this area is the CRISPR-Cas technology, which has been widely adopted as a versatile laboratory tool. The European Medicines Agency and
Genes
Researchers at Johns Hopkins University have made a groundbreaking discovery in the development of a new gene therapy approach for treating sickle cell disease. The innovative treatment utilizes nanoparticles to deliver targeted gene correction directly to bone marrow cells, reducing the complexity and side effects associated with current therapies. This
Genetic research
Researchers at the Department of Medical Oncology have made significant progress in identifying gene expression signatures associated with angiogenesis and hypoxia pathways that can predict the response to bevacizumab/erlotinib treatment in nonsquamous advanced non-small cell lung cancer patients. The study analyzed biopsy samples from 42 patients and found that
Genetic research
Research from Seoul National University has demonstrated the effectiveness of a dual gene therapy approach in inhibiting the growth of tumors. The therapy, which combines a herpes simplex virus-thymidine kinase (HSV-TK) gene with a small hairpin RNA (shRNA) specific for mammalian target of rapamycin (mTOR), has shown promise as a
Genetic research
Current research conducted at the University of Illinois has shed light on the dynamic changes that occur in dairy cows during the transition period from pregnancy to lactation. The study focused on the ruminal epithelium, examining the changes in gene expression, microbiota, and systemic biomarkers of metabolism and inflammation. The
