Description
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.- Features leading experts in the field of RNA therapeutics, spanning all classes of RNA therapies- Provides a detailed examination of approved RNA therapies and lessons learned from failed therapeutics- Covers all aspects of therapeutic discovery and preclinical development, as well as clinical translation, manufacturing and regulatory aspects
Table of Contents
SECTION 1 The world of RNA therapeutics: Available RNA tools to modulate cellular processes1. Targeting chromatin: Transcriptional gene activation (saRNA)2. Argonaute and TNRC6, partners in RNAi3. Targeted RNA therapeutics for treatment of cancer and immunomodulation4. Extrahepatic delivery of RNA to immune cellsSECTION 2 Learning from history5. Aptamer-based protein inhibitors6. Lessons learned from developing an oligonucleotide drug for a rare diseaseSECTION 3 Delivery7. Nucleoside modifications of in vitro transcribed mRNA to reduce immunogenicity and improve translation of prophylactic and therapeutic antigens8. SOMAmer reagents and the SomaScan platform: Chemically modified aptamers and their applications in therapeutics, diagnostics, and proteomicsSECTION 4 From bench to bedside9. CMC and regulatory aspects of oligonucleotide therapeutics10. CMC: Regulatory landscape11. CMC and manufacturingSECTION 5 From bench to bedside12. RNA delivery for cancer gene therapy13. Combinatorial RNA therapies in cancer immunotherapy: Challenges and directions
