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Full 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.
Contents
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
