• TNA - Therapeutic Nucleic Acids
• 대부분 극성을 띈다.
• Aptamer, SELEX
• ASO - <50bp nucleic acids
• Second generation ASO - Phosphorothioate ASO - 더 안정적
• Locked NA, peptide NA, morpholino
• FDA approved - Fomivirsen(1st), Mipomersen(2nd)
• RNA + morpholino = RNA inhibition
• Fomivirsen
  • CMV retinitis
  • CIBAVision
  • S/E) Bulls eye - reversible
  • 요즘은 AIDS에서 CMV retinitis 잘 발생 안하여 상품성 저하됨.
• Mipomersen
  • inhibitor of apolipoprotein B-100
• Gene delivery
• Gendicine - 재조합된 adenovirus
• 불확실한 long term effect - 15-year follow up - Major challenges
• Layer-by-layer
• HBV protein
• RISC - shRNA - siRNA -> getting rid of mRNA
• microRNA
• miRNA-122
  • expressed in hepatocytes - HCV
  • proliferation of HCV
• NOX-A12 (olaptesed pegol) 45-nucleotide-long RNA aptamer - CXCL12 / SDF-1 - a key regulatory chemokine
• circular RNAs - stable
• Conclusions
  • Genome decoding -> facilitation of TNA development
• Questions
  • 농도 측정 어떻게?
  • CRISPR