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Modifying immune responses to adeno-associated virus vectors by capsid engineering
Molecular Therapy: Methods & Clinical Development, 2023 De novo immune responses are considered major challenges in gene therapy. With the aim to lower innate immune responses directly in cells targeted by adeno-associated virus (AAV) vectors, we equipped the vector capsid with a peptide known to interfere ...
Martin Bentler +13 more
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Strategies of AAV capsid engineering for targeted delivery to brain, muscle, and retina
Frontiers in Molecular BiosciencesAdeno-associated virus (AAV) vectors are widely used for in vivo gene delivery to the central nervous system (CNS), muscle, and retina, but many clinically used capsids show limited potency in human tissues, necessitating high systemic doses that ...
Xinyuan Xu
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Cell Reports, 2018 Summary: The adeno-associated virus (AAV) vector is a preferred delivery platform for in vivo gene therapy. Natural and engineered variations of the AAV capsid affect a plurality of phenotypes relevant to gene therapy, including vector production and ...
Anna C. Maurer +5 more
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Overcoming Immunological Challenges Limiting Capsid-Mediated Gene Therapy With Machine Learning
Frontiers in Immunology, 2021 A key hurdle to making adeno-associated virus (AAV) capsid mediated gene therapy broadly beneficial to all patients is overcoming pre-existing and therapy-induced immune responses to these vectors.
Anna Z. Wec +6 more
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Completion of the AAV Structural Atlas: Serotype Capsid Structures Reveals Clade-Specific Features
Viruses, 2021 The capsid structures of most Adeno-associated virus (AAV) serotypes, already assigned to an antigenic clade, have been previously determined. This study reports the remaining capsid structures of AAV7, AAV11, AAV12, and AAV13 determined by cryo-electron
Mario Mietzsch +6 more
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Pathogens, 2022 Parvoviruses are a diverse family of small, non-enveloped DNA viruses that infect a wide variety of species, tissues and cell types. For over half a century, their intriguing biology and pathophysiology has fueled intensive research aimed at dissecting ...
Jonas Becker, Julia Fakhiri, Dirk Grimm
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A versatile toolkit for overcoming AAV immunity
Frontiers in Immunology, 2022 Recombinant adeno-associated virus (AAV) is a promising delivery vehicle for in vivo gene therapy and has been widely used in >200 clinical trials globally.
Xuefeng Li +5 more
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Chemical modification of AAV9 capsid with N-ethyl maleimide alters vector tissue tropism
Scientific Reports, 2023 Although more adeno-associated virus AAV-based drugs enter the clinic, vector tissue tropism remains an unresolved challenge that limits its full potential despite that the tissue tropism of naturally occurring AAV serotypes can be altered by genetic ...
Patrick L. Mulcrone +6 more
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AAV Engineering for Improving Tropism to the Central Nervous System
Biology, 2023 Adeno-associated virus (AAV) is a non-pathogenic virus that mainly infects primates with the help of adenoviruses. AAV is being widely used as a delivery vector for in vivo gene therapy, as evidenced by five currently approved drugs and more than 255 ...
Muhammad S. Ghauri, Li Ou
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Structural Insights into the Human Astrovirus Capsid
Viruses, 2021 Astroviruses (AstVs) are non-enveloped, positive single-stranded RNA viruses that cause a wide range of inflammatory diseases in mammalian and avian hosts.
Matthew Ykema, Yizhi J. Tao
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