Termin realizacji zamówienia: ok. 16-18 dni roboczych.
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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