RNA Therapeutics: The Evolving Landscape of RNA Therapeutics

ISBN-13: 9780128215951 / Angielski / Miękka / 2022 / 474 str.

Paloma H. Giangrande; Vittorio Defranciscis; John J. Rossi

Spis treści

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