caa a22 4500 475845900 CHVBK 20180406123914.0 cr unu---uuuuu 170329e20000901xx s 000 0 eng 10.1007/PL00000628 doi (NATIONALLICENCE)springer-10.1007/PL00000628 Synthetic oligonucleotides as RNA mimetics: 2′-modified RNAs and N3′→P5′ phosphoramidates [Elektronische Daten] [M. Egli, S. M. Gryaznov*] Abstract.: Significant interest in synthetic DNA and RNA oligonucleotides and their analogues has marked the past two decades of research in chemistry and biochemistry. This attention was largely determined by the great potential of these compounds for various therapeutic applications such as antisense, antigene and ribozyme-based agents. Modified oligonucleotides have also become powerful molecular biological and biochemical research tools that allow fast and efficient regulation of gene expression and gene functions in vitro and in vivo. These applications in turn are based on the ability of the oligonucleotides to form highly sequence-specific complexes with nucleic acid targets of interest. This review summarizes recent advances in the design, synthesis, biochemical and structural properties of various RNA analogues. These comprise 3′-modified oligonucleotide N3′→P5′ phosphoramidates, analogues with modifications at the 2′-position of nucleoside sugar rings, or combinations of the two. Among the properties of the RNA minetics reviewed here are the thermal stability of their duplexes and triplexes, hydrolytic resistance to cellular nucleases and biological activity in in vitro and in vivo systems. In addition, key structural aspects of the complexes formed by the RNA analogues, including interaction with water molecules and ions, are analyzed and presented. Birkhäuser Verlag Basel,, 2000 Key words. RNA mimetic nationallicence oligonucleotide N3′→P5′ phosphoramidate nationallicence sugar-base modification nationallicence structure nationallicence thermal stability nationallicence Egli M. Department of Molecular Pharmacology and Biological Chemistry and The Drug Discovery Program, Northwestern University Medical School, Chicago (Illinois 60611, USA), US aut Gryaznov* S. M. Geron Corporation, Nucleic Acids Chemistry, 230 Constitution Drive, Menlo Park (California 94025, USA), Fax +1 650 473 7750, e-mail: sgryaznov@geron.com, US aut https://doi.org/10.1007/PL00000628 text/html Onlinezugriff via DOI 1 research-article jats NATIONALLICENCE 856 40 https://doi.org/10.1007/PL00000628 text/html Onlinezugriff via DOI NATIONALLICENCE 700 1- Egli M. Department of Molecular Pharmacology and Biological Chemistry and The Drug Discovery Program, Northwestern University Medical School, Chicago (Illinois 60611, USA), US aut NATIONALLICENCE 700 1- Gryaznov* S. M. Geron Corporation, Nucleic Acids Chemistry, 230 Constitution Drive, Menlo Park (California 94025, USA), Fax +1 650 473 7750, e-mail: sgryaznov@geron.com, US aut Metadata rights reserved Springer special CC-BY-NC licence nationallicence BK010053 XK010053 XK010000 NATIONALLICENCE NATIONALLICENCE NL-springer