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...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
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MDPI AG
2023-05-01
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| Series: | International Journal of Molecular Sciences |
| Subjects: | |
| Online Access: | https://www.mdpi.com/1422-0067/24/11/9455 |
| _version_ | 1797597440641073152 |
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| author | Samuel A. Wickline Kirk K. Hou Hua Pan |
| author_facet | Samuel A. Wickline Kirk K. Hou Hua Pan |
| author_sort | Samuel A. Wickline |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-03-11T03:06:06Z |
| format | Article |
| id | doaj.art-e320f6d1da764936a5e82108c39afe5a |
| institution | Directory Open Access Journal |
| issn | 1661-6596 1422-0067 |
| language | English |
| last_indexed | 2024-03-11T03:06:06Z |
| publishDate | 2023-05-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | International Journal of Molecular Sciences |
| spelling | doaj.art-e320f6d1da764936a5e82108c39afe5a2023-11-18T07:59:15ZengMDPI AGInternational Journal of Molecular Sciences1661-65961422-00672023-05-012411945510.3390/ijms24119455Peptide-Based Nanoparticles for Systemic Extrahepatic Delivery of Therapeutic NucleotidesSamuel A. Wickline0Kirk K. Hou1Hua Pan2Division of Cardiology, Department of Medical Engineering, University of South Florida, Tampa, FL 33602, USADepartment of Ophthalmology, Stein and Doheny Eye Institutes, University of California, Los Angeles, CA 90095, USADivision of Rheumatology, Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110, USAPeptide-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.https://www.mdpi.com/1422-0067/24/11/9455nanoparticlespeptide polyplexesRNAsiRNAmRNAnucleic acids |
| spellingShingle | Samuel A. Wickline Kirk K. Hou Hua Pan Peptide-Based Nanoparticles for Systemic Extrahepatic Delivery of Therapeutic Nucleotides International Journal of Molecular Sciences nanoparticles peptide polyplexes RNA siRNA mRNA nucleic acids |
| title | Peptide-Based Nanoparticles for Systemic Extrahepatic Delivery of Therapeutic Nucleotides |
| title_full | Peptide-Based Nanoparticles for Systemic Extrahepatic Delivery of Therapeutic Nucleotides |
| title_fullStr | Peptide-Based Nanoparticles for Systemic Extrahepatic Delivery of Therapeutic Nucleotides |
| title_full_unstemmed | Peptide-Based Nanoparticles for Systemic Extrahepatic Delivery of Therapeutic Nucleotides |
| title_short | Peptide-Based Nanoparticles for Systemic Extrahepatic Delivery of Therapeutic Nucleotides |
| title_sort | peptide based nanoparticles for systemic extrahepatic delivery of therapeutic nucleotides |
| topic | nanoparticles peptide polyplexes RNA siRNA mRNA nucleic acids |
| url | https://www.mdpi.com/1422-0067/24/11/9455 |
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