Therapeutic Application and Structural Features of Adeno-Associated Virus Vector
Abstract
:1. Introduction
2. Genes and Genomic Structure of AAV
3. Serotype of AAVs
4. Efficacy of Gene Therapy by AAV
5. AAV Infection Mechanisms
6. Production of Recombinant AAV (rAAV)
7. Safety of AAV Vector
8. Transcription and Serotypes of VP Variants
9. Structural Features and Dynamics of AAV Particles
10. Structurally Dynamic Molecules and Hydrogen/Deuterium Exchange–Mass Spectrometry
11. Full and Empty Particles and Secretion of AAV
12. Novel AAV Vectors and Tropisms
12.1. Screening of Improved AAV Vector
12.2. Viral Packaging of Capsid Library
13. AAV-Based Therapies in Clinical Trials
14. Conclusions
Author Contributions
Funding
Conflicts of Interest
References
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Serotypes | Origin | Primary Receptor | Secondary Receptor | Natural Tropism | Clinical Trials |
---|---|---|---|---|---|
AAV1 | NHP | Sialic acid | AAVR | Muscle, CNS, heart, liver, lung | None |
AAV2 | Human | HSPG | Integrin, FGFR, HGFR, LamR | Heart, CNS, liver, lung, retina | Pomoe disease, Parkinson’s disease, hemophilia |
AAV3 | NHP | HSPG | LamR, FGFR, HGFR, AAVR | Liver | None |
AAV4 | NHP | Sialic acid | Unknown | Retina, lungs, kidney | None |
AAV5 | Human | Sialic acid | PDGFR, AAVR | Retina, CNS, liver | Hemophilia |
AAV6 | Human | HSPG, sialic acid | EGFR, AAVR | Heart, liver, muscle, retina | Hemophilia, mucopolysaccharidosis |
AAV7 | NHP | Unknown | Unknown | Liver | None |
AAV8 | NHP | Unknown | LamR, AAVR | Muscle, heart, CNS, liver | Eye disease, hemophilia, myopathy |
AAV9 | Human | Galactose | LamR, AAVR | Heart, CNS, liver | Muscular diseases, pompe disease, Danon disease |
AAV10 | NHP | Unknown | Unknown | Muscle, myoblast tissue | None |
AAV11 | NHP | Unknown | Unknown | Muscle, myoblast tissue | None |
AAV12 | NHP | Unknown | Unknown | Salivary glands, muscle | None |
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© 2024 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
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Matsuzaka, Y.; Yashiro, R. Therapeutic Application and Structural Features of Adeno-Associated Virus Vector. Curr. Issues Mol. Biol. 2024, 46, 8464-8498. https://doi.org/10.3390/cimb46080499
Matsuzaka Y, Yashiro R. Therapeutic Application and Structural Features of Adeno-Associated Virus Vector. Current Issues in Molecular Biology. 2024; 46(8):8464-8498. https://doi.org/10.3390/cimb46080499
Chicago/Turabian StyleMatsuzaka, Yasunari, and Ryu Yashiro. 2024. "Therapeutic Application and Structural Features of Adeno-Associated Virus Vector" Current Issues in Molecular Biology 46, no. 8: 8464-8498. https://doi.org/10.3390/cimb46080499