Surface modification of lipid nanoparticles for gene therapy.
J Gene Med
; 26(1): e3642, 2024 Jan.
Article
em En
| MEDLINE
| ID: mdl-38043928
ABSTRACT
Gene therapies have the potential to target and effectively treat a variety of diseases including cancer as well as genetic, neurological, and autoimmune disorders. Although we have made significant advances in identifying non-viral strategies to deliver genetic cargo, certain limitations remain. In general, gene delivery is challenging for several reasons including the instabilities of nucleic acids to enzymatic and chemical degradation and the presence of restrictive biological barriers such as cell, endosomal and nuclear membranes. The emergence of lipid nanoparticles (LNPs) helped overcome many of these challenges. Despite its success, further optimization is required for LNPs to yield efficient gene delivery to extrahepatic tissues, as LNPs favor accumulation in the liver after systemic administration. In this mini-review, we provide an overview of current preclinical approaches in that LNP surface modification was leveraged for cell and tissue targeting by conjugating aptamers, antibodies, and peptides among others. In addition to their cell uptake and efficiency-enhancing effects, we outline the (dis-)advantages of the different targeting moieties and commonly used conjugation strategies.
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Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Nanopartículas
/
Lipídeos
Idioma:
En
Ano de publicação:
2024
Tipo de documento:
Article