From: Nonviral delivery systems for antisense oligonucleotide therapeutics
Delivery systems | Administration routs | Targeted diseases | Nanocarriers components | Particle size | Key observations | Ref |
---|---|---|---|---|---|---|
Biodegradable lipid nanoparticles | Intravenous injection | PCSK9 in liver | 306-O12B-3, DOPE, PEG, cholesterol, ASO | 150–500 nm | ASO/LNP complexes reduce the total PCSK9 protein and serum cholesterol level with no hepatotoxicity or nephrotoxicity | [47] |
Lipid nanoparticles | Intravenous injection | Metastatic renal cancer | DOTAP, soyPC, TPGS, folate-PEG-DSPE, ASO | 108.6 ± 5.8 nm | Folate receptor-targeted lipid-albumin nanoparticles augment cell uptake rate and prolong the half-life of ASO | [48] |
Lipid nanoparticles | Intravenous injection | Lung cancer | DODMA, egg PC, cholesterol, T7-PEG-DSPE, PEG-DMG, ASO | 139.4 ± 7.6 nm | T7-conjugated CO-ASOs-LNPs exhibit excellent colloidal stability and produce superior antitumor activity | [49] |
Lipid nanoparticles | Intravenous injection | Acute myelogenous leukemia | Cholesterol, DDAB, PEI, TPGS, Tf, DOTAP, DSPE-PEG2000-Mal, ASO | 133.4 ± 7.6 nm | Transferrin-conjugated lipid nanoparticles augment cell uptake rate significantly | [50] |
Lipid nanoparticles | Intravenous injection | Hepatocellular carcinoma | Neutral cytidinyl lipid, cystine skeleton cationic lipid, DSPE-PEG, ASO | 139.0 ± 9.2 nm | Mix/CT102 nanoparticles exhibit a predominant accumulation capacity in liver tissue | [51] |
Cationic liposomes | NA | Prostate cancer | DSPE-PEG2000-Mal, Cholesterol, DOTAP, Phosphatidylcholine, Trastuzumab, ASO | 127 – 154 nm | ASO liposomes are more effective than free ASO to penetrate 2D and 3D spheroid models | [55] |
Cationic elastic liposomes | Cutaneous administration | Atopic dermatitis | DOTAP, Sodium cholate, ASO | Over 200Â nm | IL-13 ASO/cationic elastic liposomes dramatically suppress IL-13 production (by up to 70% of free ASO) | [56] |
Cationic liposomes | Injection | Candida albicans infection | DOTAP, DOPC; DOPE, MO, ASO | 40 – 80 nm | DOTAP-based lipoplexes inhibit Candida albicans filamentation up to 60% after 72 h | [57] |
Hydrogel liposomes | Subcutaneous administration | NA | Lipid-oligonucleotides, hydrogels, ASO | 14Â nm | Hydrogel-based liposomes prolong ASO release and enhance its stability | [58] |
Lipid nanoparticles | Peritumoural injection | Subcutaneous tumor | Cytidinyl lipid, cationic lipid, ASO | 236 ± 7.9 nm | Cytidinyl-lipid combined with a cationic lipid exhibits high encapsulation efficiency for ASO | [59] |
Lipid nanoparticles | Intravenous injection | Acute myelogenous leukemia | DOTAP, DOPE, TPGS, Cholesterol, DOC, PEI, ASO | 93 ± 18 nm | CD33-targeted lipid nanoparticles show a 15-fold reduction in the IC50 of an antileukemic drug | [60] |