Research on gene therapy or medicinal product with GMO

A medicinal product for gene therapy is a medicinal product which has been produced by means of a series of manufacturing techniques aimed at the in vivo or ex vivo transfer of prophylactic, diagnostic or therapeutic gene (a nuclein acid) to human or animal cells and the following expression in vivo. The expression occurs during the gene transfer with the aid of an administration system, the so-called ‘vector’, which can have a viral or non-viral origin.

Genetically modified organisms (GMO) are all organisms of which the genetic make-up has been altered though not naturally by way of reproduction and/or natural recombination. An organism is described as any biological entity which can replicate or transfer genetic material.

Gene therapy can be divided into somatic cell therapy and germ line gene therapy, depending on the cells in which the genetic material is altered. In the case of somatic gene therapy the genetic material of human cells are altered. Such alterations cannot be genetically transferred. In the case of germ line gene therapy the genetic material of gametes are altered.  The alterations are transferred to the offspring and are therefore genetically transferred. This form of gene therapy is prohibited in the Netherlands.