The Future of Tissue-Targeted Lipid Nanoparticle-Mediated Nucleic Acid Delivery
Abstract
:1. Introduction
2. LNP Chemistry, Formulation and Background
3. Inherent LNP Liver Tropism
LNP Targeting Components or Properties | Physicochemical Properties (DLS Size, PDI and Zeta Potential) | Route of Administration | Payload and Indication | Model | Tissue/Cell Type Specificity | Ref. |
---|---|---|---|---|---|---|
50 mol% 1,2-dioleoyl-3-trimethylammonium-propane (DOTAP) | 113 nm 0.22 PDI −0.52 mV | i.v. | human Erythropoietin, mouse Interleukin-10, mouse Klotho, Luciferase and Cre mRNA, and Cas9 mRNA/sgTom1 * | 18–20 g male C57BL/6 mice; (age and sex not specified) B6.Cg-Gt(ROSA)26Sortm9(CAG-tdTomato)Hze/J mice (also known as Ai9 or Ai9(RCL-tdT) mice | hepatocyte uptake | [17] |
30 molar% 1,2-dioleoyl-sn-glycero-3-phosphate (18PA) | 142 nm 0.13 PDI −2.11 mV | i.v. | human Erythropoietin, mouse Interleukin-10, mouse Klotho, Luciferase and Cre mRNA, and Cas9 mRNA/sgPTEN and Cas9 mRNA/sgTom1 * | 18–20 g male C57BL/6 mice; (age and sex not specified) B6.Cg-Gt(ROSA)26Sortm9(CAG-tdTomato)Hze/J mice (also known as Ai9 or Ai9(RCL-tdT) mice | hepatocyte uptake | [17] |
20 molar% 1,2-dioleoyl-3-dimethylammonium-propane (DODAP) | 12 nm 0.18 PDI (ZP not specified) | i.v. | human Erythropoietin, mouse Interleukin-10, mouse Klotho, Luciferase and Cre mRNA, and Cas9 mRNA/sgPCSK9 and Cas9 mRNA/sgTom1 * | 18–20 g male C57BL/6 mice; (age and sex not specified) B6.Cg-Gt(ROSA)26Sortm9(CAG-tdTomato)Hze/J mice (also known as Ai9 or Ai9(RCL-tdT) mice | hepatocyte uptake | [17] |
50:10:38.5:1.5% mole ratios DLin-KC2 DMA:DSPC:Cholesterol: DMG-PEG2000, with N:P molar ratio = 2, imparting negative charge | 75 nm (PDI not specified) −10 mV | i.m. | Luciferase mRNA * | 8-week-old female Balb/c mice | greater hepatic distribution following i.m. administration | [4] |
50:23.5:6.5:20% mole ratios 7C1 **:C14PEG2K:18:1 Lyso PC 60:10:25:5% mole ratios 7C1 **:C14PEG2K:DOPE | 20–200 nm (PDI not specified) (ZP not specified) | i.v. | ICAM-2 siRNA, Cre mRNA, CRISPR-Cas9 mRNA and ICAM-2 sgRNA * | 5–12-week-old (sex not specified) LSL-Tomato, C57BL/6J, and constitutive SpCas9 mice | lung, spleen, liver and kidney endothelial cell uptake | [37] |
Endogenous absorption of apoE to neutral LNP | 64.5 nm (PDI not specified) (ZP not specified) | i.v. | Factor VII siRNA * | 6–8-week-old female C57Bl/6, ApoE−/− and LDLR−/− mice | hepatocyte uptake | [29] |
N-acetylgalactosamine (GalNAc) ligand | 69.4 nm (PDI not specified) (ZP not specified) | i.v. | Factor VII siRNA * | 6–8-week-old female C57Bl/6 and ASGR2−/− mice | hepatocyte uptake | [29] |
Plasmalemma vesicle-associated protein (PV1) | 70 nm, 0.104 PDI and 160 nm, 0.150–0.240 PDI (ZP not specified) | i.v. | Luciferase mRNA, Cy5-mRNA * | 5-week-old female Balb-c mice | lung uptake | [7] |
Anti-Ly6c mAbs | 70 nm (PDI not specified) (ZP not specified) | i.v. | Luciferase or IL-10 mRNA; treatment of inflammatory bowel disease | Colitis was induced in: 12-week-old female C57BL/6 mice using dextran sodium sulfate | leukocyte uptake | [38] |
15–20 mol% C18PEG2000:80 mol% 7C1 **:0.1–10 mol% cholesterol | 45–50 nm <0.2 PDI (ZP not specified) | i.v. | ICAM-2 siRNA, ICAM-2 targeting sgRNA * | 5–12-week-old (sex not specified) C57BL6/j and constitutive SpCas9 mice | bone marrow endothelial cell | [39] |
Anti-CD4 antibody | 129 nm 0.12 PDI −10 mV | i.v. | Cy5-labeled siRNA and CD45 siRNA * | 6–8-week-old (sex not specified) C57BL6/j mice | T cells | [9] |
Anti-CD4 antibody | 88 nm 0.1 PDI (ZP not specified) | i.v. | Cre recombinase-encoding mRNA * | (age not specified) (sex not specified) Ai6 (RCL-ZsGreen) mice on C57BL/6J | Splenic and lymph node T cells | [10] |
Adamantane-constrained lipid | 20–200 nm 0.20–0.23 PID (ZP not specified) | i.v. | GFP siRNA * | 5–8-week-old female C57BL/6-Tg(UBC-GFP)30Scha/J, ‘GFP mice’ | splenic T cells | [40] |
Anti-CD29 antibody | 66–75 nm 0.10–0.16 PDI (ZP not specified) | i.v. | PLK1 siRNA; treatment of disseminated bone marrow mantle cell lymphoma xenograft | 8-week-old female C.B-17/IcrHsd-Prkdc scid mice | mantle cell lymphoma | [8] |
Cholesterol oleate | 22–115 nm (PDI not specified) (ZP not specified) | i.v. | ICAM-2 siRNA, GFP-targeted sgRNA * | 5–8-week-old female C57BL/6J and C57BL/6-Tg(UBC-GFP)30Scha/J, ‘GFP mice’ | hepatic endothelial cells | [41] |
~30 nm, negatively charged LNP | 34 nm 0.242 PDI −12 mV | s.c. | DiD-labeled LNP (no nucleic acid) * | 7–9-week-old female C57BL/6J mice | CD8+ dendritic cells/lymph node | [14] |
35:5:55:5% mole ratios 7C1 **:Cholesterol:C14PEG2000:DOTAP | 40 nm (PDI not specified) (ZP not specified) | nebulization | Therapeutic membrane-anchored FI6 antibody mRNA, H1N1 influenza model | 6–8-week-old female BALBc mice | lung | [42] |
~150 nm size, ~0.5% PEG density | 150 nm <0.1 PD (ZP not specified) | intravitreal and subretinal injection | Cre, mCherry, luciferase mRNA * | 1–6 months old male and female Albino BALB/c, Ai9, apoE−/−, Mertk−/− and C57BL6 mice | optic nerve, trabecular meshwork, retinal pigment epithelium, Muller glia | [43] |
Ionizable lipids with low pKa and unsaturated hydrocarbon chains | 83–229 nm 0.09–0.28 PDI (ZP not specified) | subretinal injection | Luciferase, EGFP, mCherry mRNA * | 1–4 months old male and female Albino BALB/c mice | retinal pigment epithelium | [44] |
Oxidized cholesterol | ~80 nm 0.16 PDI (ZP not specified) | i.v. | Cre mRNA * | 5–8-week-old (sex not specified) Ai14 Lox-Stop-Lox-tdTomato and C57BL/6J mice | hepatic endothelial and Kupffer cells | [30] |
Anti PECAM-1 antibody | 103 nm 0.195 PDI −4.1 mV | i.v. | Luciferase mRNA * | (age not specified) (sex not specified) C57BL/6 mice | lung vascular endothelial and immune cells | [11] |
Adamantyl-constrained lipid | 100 nm (PDI not specified) (ZP not specified) | i.v. | Cre mRNA * | (age not specified) (sex not specified) Ai14 Lox-Stop-Lox-tdTomato mice | hepatic Kupffer cells | [31] |
ApoE opsonization | 55 nm 0.058 PDI (ZP not specified) | intracranial | PTEN, luciferase and GRIN1 siRNA * | 26–30-day-old (sex not specified) Sprague Dawley rats | CNS neurons | [45] |
CH6 osteoblast-specific aptamer | 84 nm (PDI not specified) (ZP not specified) | i.v. |
osteogenic pleckstrin homology domain-containing family O member 1 (Plekho1) siRNA; treatment of impaired bone formation (e.g., osteoporosis) | 6-month-old female Sprague Dawley rats | osteoblasts | [46] |
Mannose-cholesterol | ~140 nm >0.2 PDI (ZP not specified) | i.d. | Influenze hemagglutanin saRNA; H1N1 influenza vaccine | 6−8-week-old female BALB/c mice | dendritic cells | [12] |
Mannose-PEG-DSPE | ~100 nm (PDI not specified) (ZP not specified) | i.v. | Cre mRNA and FVIII siRNA * | 7–10-week-old female C57BL/6 mice; 8-week-old (sex not specified) Lox-Stop-Lox-tdTomato | hepatic endothelial cells | [13] |
EGFR-antibody | 79 nm 0.085 PDI 7.7 mV | i.p. | Cas9 mRNA, (polo-like kinase) PLK1 sgRNA; disseminated ovarian cancer | 8-week-old female Hsd: Athymic Nude-Foxn1nu mice with OV8 ovarian cancer peritoneal xenograft | disseminated ovarian cancer | [47] |
DEC205-antibody | 90–130 nm 0.12–0.20 PDI (ZP not specified) | r.o. | CD40, CD80 and CD86 siRNA | 6–12-week-old (sex not specified) C57BL/6 mice, inhibition of mixed lymphocyte response to LPS | CD8 alpha+ dendritic cells | [48] |
CD4-antibody | 88 nm 0.1 PDI (ZP not specified) | i.v. | Luciferase and Cre mRNA * |
(age not specified) male and female C57BL/6 and Ai6 (RCL-ZsGreen) mice on C57BL/6J background | CD4+ T cells | [10] |
CD5-antibody | 80 nm 0.02–0.06 PDI (ZP not specified) | i.v. | CAR mRNA against fibroblast activation protein and Cre mRNA, cardiac fibrosis prevention | (age not specified) (sex not specified) C57BL/6NAi6 Cre-reporter mice (Rosa26CAG-LSL-ZsGreen) | CD5+ T cells | [49] |
4. Non-Hepatic LNP Targeting
5. Oncology and Immuno-Oncology
6. Conclusions and Future Directions
Author Contributions
Funding
Institutional Review Board Statement
Informed Consent Statement
Data Availability Statement
Conflicts of Interest
References
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Kularatne, R.N.; Crist, R.M.; Stern, S.T. The Future of Tissue-Targeted Lipid Nanoparticle-Mediated Nucleic Acid Delivery. Pharmaceuticals 2022, 15, 897. https://doi.org/10.3390/ph15070897
Kularatne RN, Crist RM, Stern ST. The Future of Tissue-Targeted Lipid Nanoparticle-Mediated Nucleic Acid Delivery. Pharmaceuticals. 2022; 15(7):897. https://doi.org/10.3390/ph15070897
Chicago/Turabian StyleKularatne, Ruvanthi N., Rachael M. Crist, and Stephan T. Stern. 2022. "The Future of Tissue-Targeted Lipid Nanoparticle-Mediated Nucleic Acid Delivery" Pharmaceuticals 15, no. 7: 897. https://doi.org/10.3390/ph15070897