Quick Take: Using Acute Optic Neuritis Trials to Assess Neuroprotective and Remyelinating Therapies in...

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:...

Quick Take: Genomic characterization of metastatic breast cancers

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...

Quick Take: Glucagon Receptor Antagonist–Stimulated α-Cell Proliferation Is Severely Restricted With Advanced Age

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...

Mavacamten shows promise for treating patients with obstructive hypertrophic cardiomyopathy

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...

Quick Take: Association Between Longitudinal Plasma Neurofilament Light and Neurodegeneration in Patients With Alzheimer...

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

Quick Take: Discovery and validation of a prognostic proteomic signature for tuberculosis progression

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

In utero administration of ivacaftor averts multiorgan disease in ferret model of cystic fibrosis...

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...

CRISPR-mediated gene activation rescues obesity caused by haploinsufficiency [PreClinical]

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...

MYBPC3 haploinsufficiency results in hypertrophic cardiomyopathy by altering myosin head configurations [PreClinical]

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...

De novo design of artificial IL-2 mimic for the treatment of cancer [PreClinical]

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...
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