@misc{castellanos-gonzalez_sadiqova_ortega-mendez_white_2022, title={RNA-based therapy for Cryptosporidium parvum infection: proof-of-concept studies}, DOI={10.1101/2022.01.11.475978}, abstractNote={Cryptosporidium is a leading cause of moderate-to-severe diarrhea in children. Nitazoxanide, the only FDA-approved treatment for cryptosporidiosis, has limited efficacy in those at highest risk for sequelae. RNA-argonaute (Ago) complexes to Cryptosporidium nucleoside diphosphate kinase (cpNDK) decreased the Cryptosporidium parvum mRNA by 95% in infected cells in vitro. Treatment of mice by oral gavage with ssRNA/Ago complexes encapsulated in lipid nanoparticles led to delivery of the complexes into intestinal epithelial cells. Treatment of C. parvum infected mice with ssRNA/Ago complexes targeting cpNDK led to the resolution of oocyst shedding in 4/5 SCID/beige mice. These results confirm the potential use of antisense therapy as an alternative approach to cryptosporidiosis treatment.}, publisher={Cold Spring Harbor Laboratory}, author={Castellanos-Gonzalez, Alejandro and Sadiqova, Aygul and Ortega-Mendez, Justine and White, A. Clinton}, year={2022}, month={Jan} }