Peptide-Based Nanoparticles for Systemic Extrahepatic Delivery of Therapeutic Nucleotides
Abstract
:1. Introduction
2. PBN Self Assembly
- (1)
- Electrostatic and peptide shape factors initiate binding of cell penetrating peptides into the major groove of siRNA, which is the locus for the maximal salt bridges, but only 2–3 peptides can bind to the major groove due to steric constraints.
- (2)
- Subsequent binding of peptides occurs at the minor groove with some arranged perpendicular to the siRNA. Peptides with greater + charge favored minor groove binding at this point.
- (3)
- As the negative surface charge on the siRNA becomes progressively screened, further aggregation of peptides coating the complex can occur by emerging hydrophobic peptide–peptide interactions, and the size of the overall siRNA-peptide complex can increase.
3. Functional Aspects of Key Amino Acids in PBN Formation
3.1. Arginine and Lysine
3.2. Histidine
3.3. Tryptophan
3.4. Proline
4. Extrahepatic Delivery
5. Endosomal Escape
6. PBN Applications In Vivo
6.1. Linear Cationic Peptide Self-Assembled Nanoparticles
6.1.1. Transportan/PepFect/NickFect
6.1.2. p5RHH
6.1.3. GALA/KALA/RALA
6.1.4. WRAP
7. Branched Peptide Self-Assembled Nanoparticles
7.1. “HK” Branched Polymers
7.2. Branched Amphiphilic Peptide Capsules (BAPCs)
7.3. B-mR9
7.4. Peptide Spiders
8. Conclusions
Author Contributions
Funding
Conflicts of Interest
References
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Linear Peptides | Basic Sequence | Charge |
---|---|---|
Transportan | GWTLNSAGYLLGKINLKALAALAKKIL | 4 |
Transportan 10 | AGYLLGKINLKALAALAKKIL | 4 |
PepFect14 | CH3(CH2)16-CONH-AGYLLGKLLOOLAAAALOOLL-NH2 | 5 |
C22-PepFect14-O | CH3(CH2)20-CONH-AGYLLGKLLOOLAOOALOOLL-NH2 | 7 |
NickFect 55 | Stearyl-AGYLLG)δ-OINLKALAALAKAIL-NH2 | 3 |
p5RHH | VLTTGLPALISWIRRRHRRHC | 5 |
KALA | WEARLARALARALARHLARALARALRACEA | 6 |
WRAP1 | LLWRLWRLLWRLWRLL | 5 |
WRAP5 | LLRLLRWWWRLLRLL | 5 |
Branched Peptides | ||
H2K4b-14 | [KHHKHHKHHKHHHK]4LYS | 56 |
BAPC | (Ac-FLIVI)2-K-K4-CO-NH2; (Ac-FLIVIGSII)2-K-K4--CO-NH2 | 5;5 |
B-mR9 | mR9: Cys-R9-Cys-R9-Cys | 18 |
“Spiders“ | [GWTLNSAGYLLGKINLKALAALAKKILC]6-PEG | 24 |
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Wickline, S.A.; Hou, K.K.; Pan, H. Peptide-Based Nanoparticles for Systemic Extrahepatic Delivery of Therapeutic Nucleotides. Int. J. Mol. Sci. 2023, 24, 9455. https://doi.org/10.3390/ijms24119455
Wickline SA, Hou KK, Pan H. Peptide-Based Nanoparticles for Systemic Extrahepatic Delivery of Therapeutic Nucleotides. International Journal of Molecular Sciences. 2023; 24(11):9455. https://doi.org/10.3390/ijms24119455
Chicago/Turabian StyleWickline, Samuel A., Kirk K. Hou, and Hua Pan. 2023. "Peptide-Based Nanoparticles for Systemic Extrahepatic Delivery of Therapeutic Nucleotides" International Journal of Molecular Sciences 24, no. 11: 9455. https://doi.org/10.3390/ijms24119455