Management of clinical risks deriving from insertional mutagenesis - Scientific guideline

This document discusses the factors contributing to genotoxicity of vector integration, the strategies to reduce the risk associated to insertional mutagenesis and the assays to evaluate vector oncogenesis at the pre-clinical and clinical level.

Keywords: Gene therapy medicinal products, insertional mutagenesis, integration

Current version

Reflection paper on management of clinical risks deriving from insertional mutagenesis

Adopted Reference Number: EMA/CAT/190186/2012 Summary:

Integrating vectors are considered the tools of choice by the scientific community when aiming at treating diseases that require life-long gene expression. These vectors insert permanently into the cell genome and once the cells replicate, the integrated sequences are transmitted to its progeny.