Table 3 Application of LNP-RNA therapeutics based on the target disease
LNP formulation | Payload | Specific organs/cells | Disease | Ref. |
|---|---|---|---|---|
5A2-SC8, cholesterol, DMG-PEG, DOPE | CRISPR/Cas9 | Bone marrow (hematopoietic stem cells, leukemic cells, and mature blood cells) | Sickle cell disease and acute myeloid leukemia | [304] |
DODAP, cholesterol, DMG-PEG, DSPC | siRNA | Liver | Target the FVII gene in the liver | [305] |
BP-lipid, cholesterol, C14PEG2000, DOPE | mRNA | Bone microenvironment and bone marrow (bone cells, cells of the hematopoietic and immune systems, fibroblasts, stromal cells, endothelial cells, monocytic lineage, B cell lineage, T cells, monocytes, granulocytes, B cells, and hematopoietic stem cells) | Skeletal diseases and age-related bone abnormalities (osteoporosis, osteoarthritis, osteomyelitis, and bone cancer) | [280] |
4A3-SC8, DOPE, Cholesterol, DMG-PEG, DOTAP | NG-ABE8e mRNA–sgR553X | Lungs | Genetic lung diseases, such as cystic fibrosis | [306] |
5A2-SC8, DOPE, cholesterol, DMG-PEG, 10% 18:1 PA | mRNA | CD4+ and CD8+ T cells in spleen | B cell lymphoma | [307] |
Sazo/TAzo lipidoid, DOPE, cholesterol, DMG-PEG | mRNA | Dendritic cells | Melanoma | [308] |
PEG-lipids, ionizable lipids, structural lipids, cholesterol | HNF4A mRNA | Liver | Liver fibrosis and cirrhosis | [309] |
246C10, DOPE, cholesterol, PEG-ceramide lipids | Cas9 mRNA and mouse AT-targeted sgRNA | Liver | Hemophilia | [310] |
2-(Dinonylamino)-1-(4-(N-(2-(dinonylamino)ethyl)-N-nonylglycyl)piperazin 1-yl)ethan-1-one, DSPC, cholesterol, PEG-DMG | hPBGD mRNA | Liver | Acute intermittent porphyria | [311] |
Dlin-MC3-DMA-based lipids, helper lipids, cholesterol, PEG-lipids | BisCCL2/5i mRNA | Liver | Liver cancer | [312] |
MC3 lipids, cholesterol or β-sitosterol, DMG PEG2000, DSPC | hsACE2mRNA | Liver | SARS-CoV-2 | [313] |
5A2-SC8, DOPE, cholesterol, PEG-lipids | Cas9 mRNA, PD-L1 sgRNA, and FAK siRNA | Liver | Liver cancer | [314] |
G0-C14, PDSA, DSPE-PEG, DMPE-PEG | p53 mRNA | Liver | Liver cancer | [315] |
DLin-MC3-DMA, DSPC, cholesterol, PEG2000-DMG | sPD-L1 mRNA | Lung | Acute respiratory distress syndrome | [316] |
306-N16B + 113-N16B, cholesterol, DOPE + DSPC, DMG-PEG2000 | Cas9 mRNA, sgRNA, and Tsc2 mRNA | Lung | LAM | [136] |
DLin-MC3-DMA, DPPC, cholesterol, DSPE-PEG | eGFP mRNA | Lung | Idiopathic pulmonary fibrosis | [317] |
5A2-SC8, DOPE, cholesterol, DMG-PEG | Cas9 mRNA, sgRNA, and donor ssDNA templates | Lung | Cystic fibrosis | [318] |
Modified PEIcompound7C1, cholesterol, DMG-PEG2000, cationic lipid DOTAP | mRNA encoded with broadly neutralizing antibody targeting hemagglutinin | Lung | H1N1 infection | [69] |
Lipids with bisphosphonate head groups | BMP-2 mRNA | Bone | Skeletal diseases | [280] |
DALs, DOPE, cholesterol, DMG-PEG | IL-12 mRNA | Tumor | Melanoma | [319] |
Ionizable cationic lipids, phosphatidylcholine, cholesterol, PEG-lipids | Rituximab mRNA | Tumor | Lymphoma | [320] |
cKK-E12, DSPC, cholesterol, PEG2000-DMG | HER2 antibody mRNA | Tumor | Breast cancer | [321] |
Anti-CD5 antibody, ionizable lipids, phosphatidylcholine, cholesterol, PEG-lipids | FAP-CAR mRNA | T-cell | Cardiac fibrosis | [261] |
Anti-Ly6cmAbs, MC3, DSPC, cholesterol, DMG-PEG, DSPE-PEG | IL-10mRNA | Leukocyte | Inflammatory bowel disease | [322] |
MC3, DSPC, cholesterol, DSPE-PEG2000-mannose | Ara h2 mRNA | LSECs | Peanut allergy | [323] |