Gene Therapy of Glioblastoma Multiforme - Clinical Experience on the Use of Adenoviral Vectors

Abstract

1.1 Definition of gene therapy According to the European Medicines Agency (EMA), a gene therapy medicinal product means “a biological medicinal product that contains an active substance, which contains or consists of a recombinant nucleic acid used in or administered to human beings with a view of regulating, replacing, adding or deleting a genetic sequence, as well as if its therapeutic, prophylactic or diagnostic effect relates to the recombinant nucleic acid sequence it contains, or to the products of genetic expression of this sequence”. Simplified, in the current meaning gene therapy is the transfer of nucleic acids to somatic cells of a patient to result in a therapeutic effect. Generally, gene therapy can be classified into two categories somatic gene therapy and germ line gene therapy. The difference between these two approaches is that in somatic gene therapy the genetic material is inserted to selected target cells, but the genetic information is not passed along to the next generation, whereas in germ line gene therapy the genetic information is passed on to the next generation. The classification of gene therapy into these two categories is of importance, since as up to day legislation allows gene therapy only to somatic cells.