Despite great progress in the treatment of AIDS, HIV-1 remains one of the major concerns as a human pathogen. One of the therapeutic strategies against viral infections is the application of catalytic ribonucleic acids (ribozymes) that can significantly reduce expression of a target gene by site-specific hydrolysis of its mRNA. In the present paper, we report a study on the activity of several variants of hammerhead ribozymes targeting a conserved region within mRNA encoding HIV-1 envelope glycoprotein gp41. On the basis of the data from in vitro assays and gene silencing in the cultured cells, we propose a new hammerhead ribozyme targeting the gp41-encoding sequence that can be potentially used as a therapeutic agent in AIDS treatment. Moreover, we demonstrate that the hydrolytic activity of the ribozyme in the intracellular environment cannot be inferred solely from the results of in vitro experiments.
Inhibition of HIV-1 gp41 expression with hammerhead ribozymes
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Agnieszka Fedoruk-Wyszomirska, Maciej Szymański, Paweł Głodowicz, Marta Gabryelska, Eliza Wyszko, William J. Estrin, Jan Barciszewski; Inhibition of HIV-1 gp41 expression with hammerhead ribozymes. Biochem J 1 October 2015; 471 (1): 53–66. doi: https://doi.org/10.1042/BJ20150398
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