Fragile X syndrome (FXS), the most common inherited cause of intellectual disability and autism, is caused by silencing of the Fmr1 gene, leading to a lack of the FMRP protein, which regulates protein synthesis in neurons. One key pathway affected by FMRP loss is the metabotropic glutamate receptor 5 (mGluR5) signaling pathway, where activation of mGluR5 leads to excessive translation of several proteins involved in synaptic plasticity.
Myocardial hypertrophy is a condition characterized by thickening of the ventricular wall and commonly associated with progression to heart failure. It develops when the heart is subjected to biomechanical stress or neurohormonal or hemodynamic stimuli.
Idiopathic pulmonary fibrosis (IPF) is a progressive disease characterized by lung scarring, fibrosis and finally respiratory failure. Currently, few treatment options are available for IPF and they only slow down disease progression and do not reverse fibrosis. There is a need for new therapeutic targets that aid in the management of the disease.
Hepatocellular carcinoma (HCC) is the third leading cause of cancer-related deaths worldwide, with few successful treatment options. HCC development is associated with established risk factors, including metabolic dysregulation, repeated insults by hepatotoxins or infection by hepatotropic viruses.
A recent study published in the journal International Immunopharmacology presents compelling preclinical evidence that NIMA-associated kinase 7 (NEK7), a key regulator of inflammation, may play a pivotal role in the pathology of depression.
A recent publication in Science Advances has uncovered NMNAT1 as a promising therapeutic target for alcohol-associated liver disease (ALD). Early ALD can be reversible, but prolonged alcohol abuse may lead to progressive steatohepatitis, fibrosis and even cirrhosis or hepatocellular carcinoma.
Diabetic retinopathy (DR) is a leading cause of vision loss in patients with diabetes, with limited therapeutic options. While current treatments focus on the proliferative stage, there is an urgent need to understand the underlying mechanisms of the early stages of DR to halt progression. Growing research suggests that activated microglia are key drivers of inflammation in DR.
Prostate cancer is one of the most common male-related cancers, and understanding the underlying mechanisms is crucial for developing new treatment strategies and avoiding resistance development.
Receptor tyrosine kinase-like orphan receptor 1 (ROR1), a receptor tyrosine kinase activated by Wnt5, is mainly expressed during fetal development and plays a crucial role in processes such as neurodevelopment and angiogenesis. ROR1 is nearly absent in most adult and pediatric tissues but is overexpressed in various malignancies, including leukemia and lung and breast cancers. Its expression has been correlated with poor clinical outcomes, and therefore, it is considered a promising cancer therapeutic target.
Esophageal cancer is a malignant disease with high incidence and mortality, where esophageal squamous cell carcinoma (ESCC) is the most common histological subtype. To improve therapeutic approaches for ESCC, it is key to explore the mechanisms behind the disease and find targeted inhibitors.
