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POSTER

Human iPSC-derived Cells as a Platform to Determine AAV Transduction Efficiency

This poster shows the potential of human, iPSC-derived cell types as a model to evaluate AAV-serotype transduction efficiency.

OVERVIEW

In vitro cellular models are a centerpiece for understanding, modifying, and quantifying the complexity of gene delivery. Induced pluripotent stem cells (iPSC) provide the ability to interrogate biologically relevant cell types in vitro. These cell types are compatible with
various gene targeting approaches, particularly adeno-associated viral (AAV) vectors. Defining AAV transduction permissibility across different iPSC-derived cell types is a necessity. Quantifying the effects of viral vector serotype, multiplicity of infection (MOI), promoter, media, and assay timing in a human-focused model system is possible with iPSC-derived cells.
These specialized cell types offer a major advancement for AAV studies of gene delivery.

HIGHLIGHTS

  • Introduction to AAVs
  • Representative images
  • Quantitative data of transduction efficiencies of AAV vectors into FCDI iCell products
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Modeling Neuroinflammatory Diseases with iPSC-derived Microglia

This poster shows functionally relevant and measurable phenotypes in these human iPSC-derived microglia may be suitable for modeling neuroinflammatory diseases and in drug-screening assays.

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POSTER

Evaluating Transduction Efficiencies of AAV Vectors into Human, iPSC-derived Cell Types

This poster shows the implementation of various technologies to evaluate the transduction efficiencies of AAV vectors into human, iPSC-derived cell types.

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Evaluating Transduction Efficiencies of AAV Vectors into Human iPSC-derived Cell Types

Overview

The differentiation of induced pluripotent stem cells (iPSC) into specialized cell types of the human body represents a major advancement for the development of biologically relevant in vitro models. These cellular systems enable “disease-in-a-dish” studies and are compatible with various gene targeting approaches, including adeno-associated viral (AAV) vectors, to directly correct the genetic mutation. However, the permissibility of AAV transduction across different iPSC-derived cell types needs to be defined. This requires the implementation of a sensitive and versatile technology to monitor transduction efficiencies and quantify the impact of various factors, including viral vector serotype, multiplicity of infection (MOI), choice of promoter, transduction media, and/or timing of transduction.

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