RNA Therapeutics: The Evolving Landscape of RNA Therapeutics provides a comprehensive overview of RNA therapeutic modalities, from bench-to-bedside, with an emphasis on the increasingly impactful areas of gene therapy, oligonucleotide therapeutics, gene editing and delivery. International leaders in the field examine RNA-based therapeutics tools that have been developed to-date to modulate cellular processes such as transcription, translation and protein function. Approved RNA-based therapies and lessons learned from failed therapies are discussed in-depth, as are evolving advances in RNA biochemical analysis, and similar advances that are enabling clinical application of RNA-based therapies.
Later sections discuss delivery technologies, remaining hurdles in research and translation, the therapy development process from the lab to the clinic, and novel RNA-based therapies currently in development.
1. RNA Therapeutics - p. iii 2. Cover - p. iii 3. Title Page - p. iii 4. Copyright Page - p. iv 5. Table Of Contents - p. iv 6. Contributors - p. xiii 7. Chapter 1 Targeting chromatin: Transcriptional gene activation (saRNA) - p. 3 8. Chapter 2 Argonaute and TNRC6, partners in RNAi - p. 17 9. Chapter 3 Targeted RNA therapeutics for treatment of cancer and immunomodulation - p. 37 10. Chapter 4 Extrahepatic delivery of RNA to immune cells - p. 57 11. Chapter 5 Aptamer-based protein inhibitors - p. 89 12. Chapter 6 Lessons learned from developing an oligonucleotide drug for a rare disease - p. 121 13. Chapter 7 Nucleoside modifications of in vitro transcribed mRNA to reduce immunogenicity and improve translation of prophylactic and therapeutic antigens - p. 141 14. Chapter 8 SOMAmer reagents and the SomaScan platform: Chemically modified aptamers and their applications in therapeutics, diagnostics, and proteomics - p. 171 15. Chapter 9 CMC and regulatory aspects of oligonucleotide therapeutics☆ - p. 263 16. Chapter 10 CMC: Regulatory landscape - p. 321 17. Chapter 11 CMC and manufacturing - p. 353 18. Chapter 12 RNA delivery for cancer gene therapy - p. 375 19. Chapter 13 Combinatorial RNA therapies in cancer immunotherapy: Challenges and directions - p. 425 20. Index - p. 451
RNA Therapeutics: The Evolving Landscape of RNA Therapeutics provides a comprehensive overview of RNA therapeutic modalities, from bench-to-bedside, with an emphasis on the increasingly impactful areas of gene therapy, oligonucleotide therapeutics, gene editing and delivery. International leaders in the field examine RNA-based therapeutics tools that have been developed to-date to modulate cellular processes such as transcription, translation and protein function. Approved RNA-based therapies and lessons learned from failed therapies are discussed in-depth, as are evolving advances in RNA biochemical analysis, and similar advances that are enabling clinical application of RNA-based therapies.
Later sections discuss delivery technologies, remaining hurdles in research and translation, the therapy development process from the lab to the clinic, and novel RNA-based therapies currently in development.
1. RNA Therapeutics - p. iii 2. Cover - p. iii 3. Title Page - p. iii 4. Copyright Page - p. iv 5. Table Of Contents - p. iv 6. Contributors - p. xiii 7. Chapter 1 Targeting chromatin: Transcriptional gene activation (saRNA) - p. 3 8. Chapter 2 Argonaute and TNRC6, partners in RNAi - p. 17 9. Chapter 3 Targeted RNA therapeutics for treatment of cancer and immunomodulation - p. 37 10. Chapter 4 Extrahepatic delivery of RNA to immune cells - p. 57 11. Chapter 5 Aptamer-based protein inhibitors - p. 89 12. Chapter 6 Lessons learned from developing an oligonucleotide drug for a rare disease - p. 121 13. Chapter 7 Nucleoside modifications of in vitro transcribed mRNA to reduce immunogenicity and improve translation of prophylactic and therapeutic antigens - p. 141 14. Chapter 8 SOMAmer reagents and the SomaScan platform: Chemically modified aptamers and their applications in therapeutics, diagnostics, and proteomics - p. 171 15. Chapter 9 CMC and regulatory aspects of oligonucleotide therapeutics☆ - p. 263 16. Chapter 10 CMC: Regulatory landscape - p. 321 17. Chapter 11 CMC and manufacturing - p. 353 18. Chapter 12 RNA delivery for cancer gene therapy - p. 375 19. Chapter 13 Combinatorial RNA therapies in cancer immunotherapy: Challenges and directions - p. 425 20. Index - p. 451
