RNA-based therapy for Cryptosporidium parvum infection: proof-of-concept studies [article]

Alejandro Castellanos-Gonzalez, Aygul Sadiqova, Justine Ortega-Mendez, A. Clinton White
2022 bioRxiv   pre-print
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
more » ... nto 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.
doi:10.1101/2022.01.11.475978 fatcat:lcwbzt2cirgnpibakqduyb72au