Streamlining gene therapy with AAVs

Streamlining gene therapy with AAVs

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Overview

Gene therapies are enabling a rapid expansion in the scope of treatable disease. Adeno-associated viruses (AAVs) are particularly advantageous delivery vehicles due to simplicity, safety, and efficacy in providing functional genes of interest.

This paper discusses critical considerations for a successful AAV discovery and development pipeline. Newer technologies and design factors are highlighted that increase both speed and safety.

Key Objectives:

AAVs are an excellent modality for introducing functional genes to specific tissues or cells.
A successful AAV discovery and development pipeline covers basic vector design, in vitro assays, in vivo biodistribution, immunogenicity, efficacy, and safety studies.
New self-silencing adenovirus technology enables the safe, efficient creation of AAVs without contaminating adenoviruses.

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