Intracellular Delivery of mRNA for Cell-Selective CRISPR/Cas9 Genome Editing using Lipid Nanoparticles.
Chembiochem
; 24(9): e202200801, 2023 05 02.
Article
in English
| MEDLINE | ID: covidwho-2242957
ABSTRACT
Messenger RNA (mRNA) is being used as part of an emerging class of biotherapeutics with great promise for preventing and treating a wide range of diseases, as well as encoding programmable nucleases for genome editing. However, mRNA's low stability and immunogenicity, as well as the impermeability of the cell membrane to mRNA greatly limit mRNA's potential for therapeutic use. Lipid nanoparticles (LNPs) are currently one of the most extensively studied nanocarriers for mRNA delivery and have recently been clinically approved for developing mRNA-based vaccines to prevent COVID-19. In this review, we summarize the latest advances in designing ionizable lipids and formulating LNPs for intracellular and tissue-targeted mRNA delivery. Furthermore, we discuss the progress of intracellular mRNA delivery for spatiotemporally controlled CRISPR/Cas9 genome editing by using LNPs. Finally, we provide a perspective on the future of LNP-based mRNA delivery for CRISPR/Cas9 genome editing and the treatment of genetic disorders.
Keywords
Full text:
Available
Collection:
International databases
Database:
MEDLINE
Main subject:
Nanoparticles
/
COVID-19
Type of study:
Experimental Studies
/
Prognostic study
Topics:
Vaccines
Limits:
Humans
Language:
English
Journal:
Chembiochem
Journal subject:
Biochemistry
Year:
2023
Document Type:
Article
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