From Nucleic Acids Sequences to Molecular Medicine

1. Front Matter

   Pages i-ix

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2. RNAissance

   • Juergen Brosius

   Pages 1-18

3. Nucleic Acids as Therapeutics

   • Saraswat Pushpendra, Pareek Arvind, Bhandari Anil

   Pages 19-45

4. Thio Effects as a Tool for Mechanistic Studies of the Cleavage of RNA Phosphodiester Bonds: The Chemical Basis

   • Mikko Ora, Tuomas Lönnberg, Harri Lönnberg

   Pages 47-65

5. Off-Target Effects and Safety Aspects of Phosphorothioate Oligonucleotides

   • Martina Stessl, Christian R. Noe, Johannes Winkler

   Pages 67-83

6. Oligonucleotide Conjugates: Rationale, Synthesis, and Applications

   • Yashveer Singh, Pierre Murat, Nicolas Spinelli, Eric Defrancq

   Pages 85-120

7. CRISPR: A Bacterial Immunity System Based on Small RNAs

   • Rolf Wagner, Ümit Pul

   Pages 121-143

8. Antisense Technology: From Unique Laboratory Tool to Novel Anticancer Treatments

   • Christine Di Cresce, Colin Way, Mateusz Rytelewski, Saman Maleki Vareki, Supritha Nilam, Mark D. Vincent et al.

   Pages 145-189

9. Antisense-Mediated Reduction of Eukaryotic Noncoding RNAs

   • Xue-hai Liang, Timothy A. Vickers, Stanley T. Crooke

   Pages 191-214

10. Antisense Oligonucleotides in the Treatment of Malignant Gliomas

    • Gerardo Caruso, Mariella Caffo, Giuseppe Raudino, Federica Raudino, Mario Venza, Francesco Tomasello

    Pages 215-246

11. Natural Antisense Transcripts Mediate Regulation of Gene Expression

    • Marco Magistri, Mohammad Ali Faghihi

    Pages 247-274

12. Activation and Deactivation of Antisense and RNA Interference Function with Light

    • Jeane M. Govan, Alexander Deiters

    Pages 275-291

13. Ribozymes as Molecular Biology Reagents

    • Sanchita Bhadra, Arti Pothukuchy, Andrew Ellington

    Pages 293-312

14. RNA Technologies for Mitochondrial Genetics

    • André Dietrich, Katarzyna Rolle, Marta Gabryelska, Eliza Wyszko, Romain Val, Maciej Szymanski et al.

    Pages 313-356

15. RNA-Based Therapies for Inherited Metabolic Diseases

    • Belén Pérez, Magdalena Ugarte, Lourdes R. Desviat

    Pages 357-370

16. RNA-Cleaving DNA Enzymes and Their Potential Therapeutic Applications as Antibacterial and Antiviral Agents

    • P. I. Pradeepkumar, Claudia Höbartner

    Pages 371-410

17. Hammerhead Ribozymes Against Virus and Viroid RNAs

    • Alberto Carbonell, Ricardo Flores, Selma Gago

    Pages 411-427

18. Suppression of Hepatitis C Viral Genome Replication with RNA-Cleaving Deoxyribozyme

    • Dal-Hee Min, Dong-Eun Kim

    Pages 429-452

19. Design of Synthetic shRNAs for Targeting Hepatitis C: A New Approach to Antiviral Therapeutics

    • Brian H. Johnston, Qing Ge

    Pages 453-473

20. The Diverse Active Sites in Splicing, Debranching, and MicroRNA Processing Around RNA Phosphodiester Bonds

    • Sourav Kumar Dey, Eduardo Paredes, Molly Evans, Subha R. Das

    Pages 475-501

Despite a half century of structural, biophysical and biochemical investigations of ribonucleic acids, they are still mysterious. RNAs stand at fertile crossroads of disciplines, integrating concepts from genomics, proteomics, dynamics as well as biochemistry and molecular biology. From 20 years it is clear, that genetic regulation of eukaryotic organisms has been misunderstood for the last years that the expression of genetic information is effected only by proteins. Basic understanding of nucleic acids has enhanced our foundation to probe novel biological functions. This is especially evident for RNA molecules whose functionality, maturation, and regulation require formation of correct secondary structure through encoded base-pairing interactions.

• RNA silencing
• from bench to bed
• molecular basis of disease
• molecular medicine
• therapeutic nucleic acids

• Institute of Chemistry/Biochemistry, Free University Berlin, Berlin, Germany

  Volker A. Erdmann

• Inst. Bioorganic Chemistry, PAN Poznan, Poznan, Poland

  Jan Barciszewski

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