Title Page
Contents
요약 6
Abstract 8
1. Introduction 9
2. Materials and methods 11
2.1. Materials 11
2.2. Synthesis of poly(benzyl-β³-LCHG) with various PEGs 11
2.3. Synthesis of butyl-poly(benzyl-β³-LCHA) 13
2.4. Aminolysis 14
2.5. MALDI-TOF mass spectrometry of butyl-poly(Bn-β³-LCHG) 15
2.6. Preparation and characterization of IVT mRNA-loaded polyplexes 15
2.7. Morphological analysis 15
2.8. Determination of logD₇.₃ of poly-β-peptide derivatives by using Alexa fluor 647 NHS ester 16
2.9. Gel retardation assay 16
2.10. mRNA transfection in cultured C2C12 cells 17
2.11. Stability assay 17
2.12. Flow cytometry analysis for determining cellular uptake 17
2.13. In vivo administration of FLuc mRNA-loaded polyplexes 17
2.14. Experiment performed by other people 18
3. Results and discussion 19
3.1. Synthesis and evaluation of poly-β-peptides and derivatives 19
3.1.1. Synthesis of poly-β-peptides 19
3.1.2. Preparation and characterization of FLuc mRNA-loaded polyplexes 34
3.1.3. Evaluation of in vitro transfection efficiency of FLuc mRNA-loaded polyplexes in cultured cells 40
3.1.4. Cellular uptake of FLuc mRNA-loaded polyplexes 43
3.1.5. Biodistribution of FLuc mRNA-loaded polyplexes 45
3.2. Synthesis of poly-α-peptides 48
3.2.1. Synthesis of poly(β-benzyl-L-aspartate) (PBLA) 48
3.2.2. Preparation and characterization of FLuc mRNA-loaded polyplexes 54
4. Conclusion 56
5. References 58