The role of lipid components in lipid nanoparticles for vaccines and gene therapy.
Adv Drug Deliv Rev
; 188: 114416, 2022 09.
Article
en En
| MEDLINE
| ID: mdl-35787388
ABSTRACT
Lipid nanoparticles (LNPs) play an important role in mRNA vaccines against COVID-19. In addition, many preclinical and clinical studies, including the siRNA-LNP product, Onpattro®, highlight that LNPs unlock the potential of nucleic acid-based therapies and vaccines. To understand what is key to the success of LNPs, we need to understand the role of the building blocks that constitute them. In this Review, we discuss what each lipid component adds to the LNP delivery platform in terms of size, structure, stability, apparent pKa, nucleic acid encapsulation efficiency, cellular uptake, and endosomal escape. To explore this, we present findings from the liposome field as well as from landmark and recent articles in the LNP literature. We also discuss challenges and strategies related to in vitro/in vivo studies of LNPs based on fluorescence readouts, immunogenicity/reactogenicity, and LNP delivery beyond the liver. How these fundamental challenges are pursued, including what lipid components are added and combined, will likely determine the scope of LNP-based gene therapies and vaccines for treating various diseases.
Palabras clave
Texto completo:
1
Base de datos:
MEDLINE
Asunto principal:
Vacunas
/
Ácidos Nucleicos
/
Nanopartículas
/
COVID-19
Límite:
Humans
Idioma:
En
Revista:
Adv Drug Deliv Rev
Asunto de la revista:
FARMACOLOGIA
/
TERAPIA POR MEDICAMENTOS
Año:
2022
Tipo del documento:
Article
País de afiliación:
Dinamarca