Adeno-associated virus (AAV) vectors: Rational design strategies for capsid engineering
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Adeno-associated virus (AAV) consists of a simple genome, infects mammalian cells, displays nonpathogenicity in humans, and spans an array of serotypes and variants bearing distinct tissue tropisms. These attributes lend AAV tremendous promise as a gene delivery vector, further substantiated by its extensive testing in human clinical trials. Rational design approaches to capsid engineering leverage current scientific knowledge of AAV to further modulate, enhance and optimize the performance of the vectors. Capsid modification strategies include amino acid point mutations, peptide domain insertions, and chemical biology approaches. Through such efforts, insights regarding AAV capsid sequence-structure-function relationships can be learned. Developments over the last 5 years in rational design-based capsid engineering approaches will be presented and discussed.
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Rational design takes in-depth biological knowledge of existing AAV serotypes to predict and refine virus function with increased capsid tropism, enhanced transduction efficiency, and reduced immune response.
“Rational design takes in-depth biological knowledge of existing AAV serotypes to predict and refine virus function with increased capsid tropism, enhanced transduction efficiency, and reduced immune response [55, 56].”
From Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside (2024) · cites this paper as [56]
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Overview of analytics needed to support a robust gene therapy manufacturing process
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Emerging Concepts in Vector Development for Glial Gene Therapy: Implications for Leukodystrophies
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Gene therapy for Parkinson’s disease: a new frontier in neurodegenerative diseases
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Highly Selective Lysine Acylation in Proteins Using a Lys‐His Tag Sequence
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Genome editing in the adrenal gland: a novel strategy for treating congenital adrenal hyperplasia
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The amazing AAV capsids: Into the structure-verse
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Structural basis for nuclear import of bat adeno-associated virus capsid protein
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Gene therapy for retinal diseases: From genetics to treatment
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Unlocking the potential of adeno-associated virus in neuroscience: a brief review
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On the road to in vivo CAR-T success: Comparing promising viral and non-viral vectors.
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Considerations for the Use of Viral Vectors in Nonhuman Primates
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Emerging drug delivery strategies for local immunomodulation in atherosclerosis
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Micro/nanomotors in biomedicine: Construction and applications
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Efficient Nonviral Stable Transgenesis Mediated by Retroviral Integrase
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Designing molecules: directing stem cell differentiation
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Adeno-associated viral vector targeted evolution for neurofibromatosis gene delivery
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Gene delivery and biomedical engineering
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- Authors
- Lee, E. J., Guenther, C. M., & Suh, J.
- Affiliations
- Rice University (US)
- Corresponding author
- Suh, Junghae
- Venue
- Current Opinion in Biomedical Engineering
- Publisher
- Elsevier BV
- Published
- 2018-09
- Volume
- 7
- Pages
- 58-63
- ISSN
- 2468-4511 (ISSN-L)
- PMC
- PMC6516759
- OpenAlex
- W2892515295
- Cited by
- 83
- References
- 52
- Citations by year
- 2026: 5 · 2025: 15 · 2024: 16 · 2023: 9 · 2022: 12 · 2021: 9 · 2020: 15 · 2019: 1 · 2018: 1
- FWCI
- 0.91
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Licence
- www.elsevier.com/tdm/userlicense/1.0/ (tdm)
- www.elsevier.com/legal/tdmrep-license (tdm)
- www.elsevier.com/open-access/userlicense/1.0/ (am, 406 d)
- doi.org/10.15223/policy-017 (stm-asf)
- doi.org/10.15223/policy-037 (stm-asf)
- doi.org/10.15223/policy-012 (stm-asf)
- doi.org/10.15223/policy-029 (stm-asf)
- doi.org/10.15223/policy-004 (stm-asf)
- Locations
- Current Opinion in Biomedical Engineering (acceptedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Science Foundation (1611044)
- National Institutes of Health (R01CA207497, R01HL138126)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Neuroscience › Neurology › Barrier Structure and Function Studies
- Keywords
- adeno-associated virus vectors, capsid engineering, rational design, gene delivery vectors, AAV serotypes, tissue tropism, point mutations, sequence-structure-function relationships
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