HIV and Ribozymes

Advances in Experimental Medicine and Biology
Robert J Scarborough, Anne Gatignol

Abstract

Ribozymes are structured RNA molecules that act as catalysts in different biological reactions. From simple genome cleaving activities in satellite RNAs to more complex functions in cellular protein synthesis and gene regulation, ribozymes play important roles in all forms of life. Several naturally existing ribozymes have been modified for use as therapeutics in different conditions, with HIV-1 infection being one of the most studied. This chapter summarizes data from different preclinical and clinical studies conducted to evaluate the potential of ribozymes to be used in HIV-1 therapies. The different ribozyme motifs that have been modified, as well as their target sites and expression strategies, are described. RNA conjugations used to enhance the antiviral effect of ribozymes are also presented and the results from clinical trials conducted to date are summarized. Studies on anti-HIV-1 ribozymes have provided valuable information on the optimal expression strategies and clinical protocols for RNA gene therapy and remain competitive candidates for future therapy.

Citations

Dec 28, 2017·Viruses·Robert J Scarborough, Anne Gatignol
Jan 30, 2018·Frontiers in Immunology·Luca Vangelista, Sandro Vento
Jan 17, 2019·Nucleic Acids Research·Xin HuangXianming Mo
Dec 23, 2020·Human Gene Therapy·Tatjana I CornuToni Cathomen
Jan 14, 2020·Molecular Therapy. Nucleic Acids·Yuhua WengXing-Jie Liang

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