Brain gene therapy with Trojan horse lipid nanoparticles.
Trends Mol Med
; 29(5): 343-353, 2023 05.
Article
in English
| MEDLINE | ID: covidwho-2275605
ABSTRACT
The COVID-19 mRNA vaccine was developed by the scalable manufacture of lipid nanoparticles (LNPs) that encapsulate mRNA within the lipid. There are many potential applications for this large nucleic acid delivery technology, including the delivery of plasmid DNA for gene therapy. However, gene therapy for the brain requires LNP delivery across the blood-brain barrier (BBB). It is proposed that LNPs could be reformulated for brain delivery by conjugation of receptor-specific monoclonal antibodies (MAbs) to the LNP surface. The MAb acts as a molecular Trojan horse to trigger receptor-mediated transcytosis (RMT) of the LNP across the BBB and subsequent localization to the nucleus for transcription of the therapeutic gene. Trojan horse LNPs could enable new approaches to gene therapy of the brain.
Keywords
Full text:
Available
Collection:
International databases
Database:
MEDLINE
Main subject:
Nanoparticles
/
COVID-19
Topics:
Vaccines
Limits:
Humans
Language:
English
Journal:
Trends Mol Med
Journal subject:
Molecular Biology
Year:
2023
Document Type:
Article
Similar
MEDLINE
...
LILACS
LIS