Modification of Lipid-Based Nanoparticles: An Efficient Delivery System for Nucleic Acid-Based Immunotherapy
Abstract
:1. Introduction
2. Modifications of Lipid-Based Nanoparticles
2.1. Types of Lipid-Based Nanoparticles
2.1.1. Liposomes
2.1.2. Lipid Nanoparticles
2.1.3. Lipid Nanoemulsions
2.1.4. Solid Lipid Nanoparticles
2.1.5. Nanostructured Lipid Carriers
2.1.6. Other Nanostructured Lipid Carriers
2.2. Development and Modification Strategies
2.2.1. Component Modification
2.2.2. Surface Modification
3. Applications of Lipid-Based Nanoparticles
3.1. LBNP in Treatment of Inherited Disease
3.2. LBNP in the Treatment of Infectious Diseases
3.3. LBNP in the Treatment of COVID-19
3.4. LBNP in the Treatment of Cancer Immunotherapy
4. Conclusions and Future Perspectives
Author Contributions
Funding
Institutional Review Board Statement
Informed Consent Statement
Data Availability Statement
Acknowledgments
Conflicts of Interest
References
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Genetic Drugs | Target | Indication | Clinical Trials Identifier or Approved Year |
---|---|---|---|
siRNA | |||
Patisiran (ALN-TTR02) | Transthyretin | Hereditary transthyretin-mediated amyloidosis | FDA-approved in 2018 |
Givosiran (ALN-AS1) | 5-Aminolevulinic acid synthase | Acute hepatic porphyria | FDA-approved in 2019 |
Lumasiran (ALN-GO1) | HAO1 | Primary hyperoxaluria type 1 | FDA-approved in 2020 |
Inclisiran (ALN-PCSSC) | PCSK9 | Hypercholesterolemia | FDA-approved in 2021 |
ALN-VSP02 | VEGF-A, KSP | Solid tumors | NCT01158079 |
ARB-001467 | HBsAg | Hepatitis B, chronic | NCT02631096 |
TKM-PLK1 | PLK1 | Adrenocortical carcinoma, hepatocellular carcinoma | NCT01437007 |
siRNA-EphA2-DOPC | EphA2 | Advanced or recurrent solid tumors | NCT01591356 |
Atu027 | Protein kinase N3 | Advanced solid cancer | NCT00938574 |
ND-L02-s0201 | HSP47 | Hepatic fibrosis | NCT03241264 |
DCR-MYC | Oncogene MYC | Solid tumors, hepatocellular carcinoma | NCT02314052 |
PRO-040201 | Apo-B | Hypercholesterolemia | NCT00927459 |
mRNA | |||
mRNA-1273 | SARS-CoV-2 | COVID-19 vaccine | FDA-approved in 2022 |
BNT162b2 | SARS-CoV-2 | COVID-19 vaccine | FDA-approved in 2021 |
mRNA-2416 | OX40L | Metastatic solid tumor, lymphoma | NCT03323398 |
mRNA-2752 | OX40L | Lymphoma, ovarian | NCT03739931 |
mRNA-1647 | 6 CMV | Cytomegalovirus infection | NCT03382405 |
Antisense oligonucleotides | |||
Mipomersen | ApoB-100 | Homozygous familial hypercholesterolemia | FDA-approved in 2013 |
Nusinersen | SMN2 gene | Spinal muscular atrophy | FDA-approved in 2016 |
Golodirsen | Dystrophin | Duchenne muscular dystrophy | FDA-approved in 2019 |
Volanesorsen | Apo-CIII | Familial chylomicronaemia syndrome | NCT02658175 |
Viltolarsen | Exon 45 | Duchenne muscular dystrophy | FDA-approved in 2020 |
Casimersen | Exon 45 | Duchenne muscular dystrophy | FDA-approved in 2021 |
Drug Delivery System | Characteristics | Application |
---|---|---|
Liposome | Spherical vesicles with lipid bilayer | Delivery of small molecule drug, oligo nucleotide |
Lipid nanoparticle | Multilamellar and faceted nano-sized vesicle with w/o/w phase | Delivery of nucleic acids for gene therapy, gene editing, and genomic engineering |
Lipid nanoemulsion | Spherical vesicles with a lipid monolayer and a lipid core | Delivery of hydrophobic or unstable drugs |
Solid lipid nanoparticle | Spherical vesicles with a core consisting of solid matrix | Delivery of Hydrophobic drugs, active lipid ingredients, and nucleic acids |
Nanostructured lipid carrier | Spherical vesicles with a core consisting of both solid and liquid lipids | Delivery of virus, non-viral nucleic acids, small molecule drugs with low aqueous solubility |
Exosome | Nano-sized lipid vesicles secreted from living cells, ranging from 30 to 200 nm, present in cell culture medium and other biological fluids | Carrier of various proteins and nucleic acids for intercellular communication |
Products | Formulation | FDA Approval Year | Mechanism | Indication |
---|---|---|---|---|
Doxil | Liposomal doxorubicin | 1995 | Topoisomerase II inhibitor | Leukemias, multiple myeloma, Hodgkin’s lymphoma, various cancers |
DaunoXome | Liposomal daunoribucin | 1996 | Topoisomerase II inhibitor | Various cancers, HIV-associated Kaposi’s sarcoma |
AmBisome | liposomal amphotericin B | 1997 | Binding to ergosterol and cause ion leakage | Invasive fungal infection |
Marqibo | Liposomal vincristine | 2012 | Tubulin inhibitor | Lymphoma, leukemia, melanoma, brain cancer |
ONIVYDE | Liposomal irinotecan | 2015 | Topoisomerase I inhibitor | Colon cancer, small-cell lung cancer |
Lipusu | Liposomal paclitaxel | 2016 | Microtubule inhibitor | Breast cancer, non-small-cell lung cancer |
Vyxeos | Liposomal daunorubicin and cytarabine | 2017 | Topoisomerase II inhibitor, antimetabolic | Acute myeloid leukemia (AML) |
ONPATTRO | Patisiran siRNA LNP | 2018 | siRNA target to transthyretin | Hereditary transthyretin-mediated amyloidosis |
GIVLAARI | Givosiran siRNA LNP | 2019 | siRNA target to 5-aminolevulinic acid synthase | Acute hepatic porphyria |
OXLUMO | Lumasiran siRNA LNP | 2020 | siRNA target to HAO1 | Primary hyperoxaluria type 1 |
Leqvio | Inclisiran siRNA LNP | 2021 | siRNA target to PCSK9 | Hypercholesterolemia |
mRNA-1273 | mRNA LNP | 2022 | Encoding SARS-CoV-2 S protein | COVID-19 vaccine |
BNT162b2 | mRNA LNP | 2021 | Encoding SARS-CoV-2 S protein | COVID-19 vaccine |
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Zhang, C.; Ma, Y.; Zhang, J.; Kuo, J.C.-T.; Zhang, Z.; **e, H.; Zhu, J.; Liu, T. Modification of Lipid-Based Nanoparticles: An Efficient Delivery System for Nucleic Acid-Based Immunotherapy. Molecules 2022, 27, 1943. https://doi.org/10.3390/molecules27061943
Zhang C, Ma Y, Zhang J, Kuo JC-T, Zhang Z, **e H, Zhu J, Liu T. Modification of Lipid-Based Nanoparticles: An Efficient Delivery System for Nucleic Acid-Based Immunotherapy. Molecules. 2022; 27(6):1943. https://doi.org/10.3390/molecules27061943
Chicago/Turabian StyleZhang, Chi, Yifan Ma, **g**g Zhang, Jimmy Chun-Tien Kuo, Zhongkun Zhang, Haotian **e, **g Zhu, and Tongzheng Liu. 2022. "Modification of Lipid-Based Nanoparticles: An Efficient Delivery System for Nucleic Acid-Based Immunotherapy" Molecules 27, no. 6: 1943. https://doi.org/10.3390/molecules27061943