Silencing Genes to Identify New Targets for Drug Development

Alejandro Castellanos-Gonzalez of the University of Texas Medical Branch in the U.S. will use their gene silencing approach involving premade complexes of protein and small RNA to identify drug targets in the Cryptosporidium parasite, which causes severe diarrhea in young children in developing countries. Their gene silencing method involves synthesizing the so-called argonaute protein that is able to cut a single gene and attaching it to a single-strand antisense RNA that is designed to target a specific gene. This method can be easily scaled up for high-throughput drug target screens. They identified 100 candidate genes that are likely to be essential for the parasite to function and are not similar to genes found in humans, making them potentially suitable as targets for drugs. They will determine whether these genes can be fully silenced using their approach, and for 20 candidate targets evaluate the effect of silencing on parasite infection in cultured cells.