Preclinical

1. Acute optic neuritis appears may be an appropriate condition to test neuroprotective and re-myelinating therapies after acute inflammation in multiple sclerosis.Evidence Rating Level:...

Several late-stage cancers have been shown to acquire genomic alterations during disease evolution. In patients with breast cancer, 20% of metastatic tumors develop ESR1...

Glucagon-containing α-cells are important for regulating glucose homeostasis along with insulin-secreting β-cells. Glucagon receptor antagonists (GRAs) hold potential as antidiabetic therapies, but there is...

1. In this open-label clinical trial, mavacamten was found to reduce mean postexercise left ventricular outflow tract gradient after 12 weeks of treatment, which...

Alzheimer disease (AD) is characterized by the accumulation of amyloid b (Ab) and tau aggregates in the brain. The progression of AD can be...

Tuberculosis (TB) is the leading cause of death by an infectious disease. Identifying affected individuals, particularly if they are asymptomatic, however, remains a challenge...

1. A G551D missense mutation in the cystic fibrosis transmembrane conductance receptor (CFTR) is a genetic cause of cystic fibrosis (CF).2. In utero administration...

1. Haploinsufficiency of Sim1 or Mc4r in the hypothalamus result in an obesity phenotype in mice and humans.2. In mice, CRISPR-mediated activation (CRISPRa) of...

1. Truncating mutations in cardiac myosin-binding protein C (MYBPC3) are a common genetic cause of hypertrophic cardiomyopathy.2. Haploinsufficiency of MYBPC3 results in cardiomyocyte hypercontractility...

1. IL-2 is a central cytokine that enhances the immune response, with considerable potential for the treatment of cancer.2. A mimic of IL-2 binds...