AAV Capsid Modification

Adeno-associated virus (AAV) is a prominent gene therapy delivery vehicle abundantly used in clinical trials worldwide. However, the restricted cell and tissue tropism of many of the serotypes limit their applicability to treating multisystem diseases. To overcome these limitations, Creative Biolabs offers high-quality AAV capsid modification services to promote the development of global customers' projects.

AAV Capsid Modification

To date, more than one hundred different AAV serotypes have been identified from different animals. Despite these remarkable achievements, experiments in vitro and in vivo have demonstrated that the AAV transduction efficiency of natural AAV serotype vectors varies significantly among cell types and different tissues. To fulfill its role as a recombinant viral expression vector, the original wild type AAV has been extensively modified to improve cell transduction and to evade the host immune response. Particularly, the capsid gene of the virus determines the properties of viral particles such as tissue tropism and antigenic properties. Capsid modification refers to the intentional modification of hypervariable loops (and possibly other regions) of the capsid to achieve a desired biological property.

Strategies for AAV vectors retargeting. Figure 1. Strategies for AAV vectors retargeting. (Alméciga-Díaz, 2011)

Currently, several techniques can be used to generate novel AAV capsids with unique, targeted tropism, including chemical modification of the virus capsid, production of hybrid capsids, peptide insertion, capsid shuffling, directed evolution, and rational mutagenesis. The large array of capsid variants generated by these strategies, along with techniques that can enhance or alter their native tropisms, provides a rapidly expanding approach for gene transfer to multiple tissues.

Services

As a leading company in the gene therapy area, Creative Biolabs is able to offer comprehensive AAV capsid modification services for worldwide researchers, including but not limited to the site-specific modification, transcapsidation, and chimeric capsid construction. With years of experience, our scientists have further refined the tropism of AAV through numerous methods, such as genetic modification of AAV Vector, adapter mediated AAV vector targeting, etc.

Especially, we can modify AAV capsid by pseudotyping or the mixing of a capsid and genome from different viral serotypes to improve AAV vector transduction efficiency as well as alter tropism. In addition, our staff has also experimented with hybrid capsids derived from multiple different serotypes to alter viral tropism.


With decades of experience, Creative Biolabs has a unique and comprehensive portfolio to support the development of cellular therapy programs from animal model selection to clinical testing support. Our advanced technology platform allows us to provide our clients with flexible, comprehensive solutions to maximize resources and optimize results. For more detailed information, please feel free to contact us.

Reference

  1. Alméciga-Díaz, C.J.; et al. (2011). Gene delivery systems: tailoring vectors to reach specific tissues. In Non-Viral Gene Therapy. IntechOpen.
For research use only. Not intended for any clinical use.

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