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RNA Therapeutics
The Evolving Landscape of RNA Therapeutics
Provides a deep examination of RNA therapeutic modalities, from bench-to-bedside, enabling new research and clinical translation
Paloma H. Giangrande (Edited by), Vittorio de Franciscis (Edited by), John J. Rossi (Edited by)
9780128215951, Elsevier Science
Paperback, published 13 April 2022
472 pages, 100 illustrations (50 in full color)
23.5 x 19 x 3 cm, 0.97 kg
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.
SECTION 1 The world of RNA therapeutics: Available RNA tools to modulate cellular processes 1. Targeting chromatin: Transcriptional gene activation (saRNA) 2. Argonaute and TNRC6, partners in RNAi 3. Targeted RNA therapeutics for treatment of cancer and immunomodulation 4. Extrahepatic delivery of RNA to immune cells SECTION 2 Learning from history 5. Aptamer-based protein inhibitors 6. Lessons learned from developing an oligonucleotide drug for a rare disease SECTION 3 Delivery 7. Nucleoside modifications of in vitro transcribed mRNA to reduce immunogenicity and improve translation of prophylactic and therapeutic antigens 8. SOMAmer reagents and the SomaScan platform: Chemically modified aptamers and their applications in therapeutics, diagnostics, and proteomics SECTION 4 From bench to bedside 9. CMC and regulatory aspects of oligonucleotide therapeutics 10. CMC: Regulatory landscape 11. CMC and manufacturing SECTION 5 From bench to bedside 12. RNA delivery for cancer gene therapy 13. Combinatorial RNA therapies in cancer immunotherapy: Challenges and directions
