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Gene Knockdown System for Cryptosporidium

William Witola of the University of Illinois in the U.S. will help develop new drugs for treating children infected with the protozoa Cryptosporidium by using a gene knockdown approach to evaluate candidate drug targets. Found in contaminated water, Cryptosporidium is the second most common cause of potentially lethal diarrhea in young children in developing countries. There are no safe and effective drugs available due largely to the lack of genetic tools for studying Cryptosporidium in the laboratory. Phosphorodiamidate morpholino oligomers (PPMOs) are small molecules that can be designed to bind and silence specific genes also in protozoa. If these genes encode for proteins critical for vital cellular functions, the PPMOs can cause death. To evaluate his approach, he will design PPMOs and test their ability to silence essential Cryptosporidium genes and thereby block chronic infection in mice.

More information about Accelerate Development of New Therapies for Childhood Cryptosporidium Infection (Round 17)

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