Niclosamide, an FDA-approved oral anthelmintic drug, has broad biological activity including anticancer, antibacterial, and antiviral properties. Niclosamide has also been identified as a potent inhibitor of SARS-CoV-2 infection in vitro, generating interest in its use for the treatment or prevention of COVID-19. Unfortunately, there are several potential issues with using niclosamide for COVID-19, including low [...]
Despite significant progress, lung cancer remains the nation’s leading cause of cancer related deaths. Recently, the Jacks Lab from MIT has unveiled a new repurposed drug candidate for a form of aggressive lung cancer called small cell lung cancer (SCLC). This exciting result comes from a 5,000 gene CRISPR knock out model in mice [...]
Rufini et al. provide an excellent case study of drug repurposing using a known drug, Etravirine, an antiretroviral used to treat human immunodeficiency (HIV)-infected patients. Here Etravirine was used to treat Friedreich’s ataxia (FRDA) an autosomal-recessive disease characterized by gait instability and loss of muscle coordination. Rufini et al. discuss the process of uncovering [...]
Arkin et al. at the University of California, San Francisco provide a tremendously insightful look at the process of small-molecule screening as a way to discover new and innovative ways to treat existing genetic diseases. This review encompasses the entire process of small-molecule screening starting with conceptualization and experimental design to hit validation and [...]
Google in collaboration with the Rubin Lab at Harvard provide a fantastic overview into high-content screening as a tool for understanding disease phenotypes. Highlighting the use of machine learning as a resource for identifying patients with spinal muscular atrophy (SMA) and those without. Many of the methods comprehensively discussed in this paper are specifically [...]
