Peptide-Based Nanoparticles for Systemic Extrahepatic Delivery of Therapeutic Nucleotides

Peptide-based nanoparticles (PBN) for nucleotide complexation and targeting of extrahepatic diseases are gaining recognition as potent pharmaceutical vehicles for fine-tuned control of protein production (up- and/or down-regulation) and for gene delivery. Herein, we review the principles and mechani...

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Bibliographic Details
Published inInternational journal of molecular sciences Vol. 24; no. 11; p. 9455
Main Authors Wickline, Samuel A, Hou, Kirk K, Pan, Hua
Format Journal Article
LanguageEnglish
Published Switzerland MDPI AG 29.05.2023
MDPI
Subjects
Amyloidosis
Gene transfer
Health aspects
Lipids
Liver
mRNA
Nanoparticles
nucleic acids
Nucleotides
peptide polyplexes
Peptides
Regulatory approval
Review
RNA
Self-assembly
siRNA
Vaccines
extrahepatic delivery
peptide-based nanoparticles
nanoparticles
RNA
endosomal escape
mRNA
siRNA
nucleic acids
peptide polyplexes
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Summary:Peptide-based nanoparticles (PBN) for nucleotide complexation and targeting of extrahepatic diseases are gaining recognition as potent pharmaceutical vehicles for fine-tuned control of protein production (up- and/or down-regulation) and for gene delivery. Herein, we review the principles and mechanisms underpinning self-assembled formation of PBN, cellular uptake, endosomal release, and delivery to extrahepatic disease sites after systemic administration. Selected examples of PBN that have demonstrated recent proof of concept in disease models in vivo are summarized to offer the reader a comparative view of the field and the possibilities for clinical application.
Bibliography:ObjectType-Article-2
SourceType-Scholarly Journals-1
ObjectType-Feature-3
content type line 23
ObjectType-Review-1
ISSN:1422-0067
1661-6596
1422-0067
DOI:10.3390/ijms24119455