The enormous progress within the last 20 years in
molecular biology and biotechnology has opened
up new possibilities for the treatment of many diseases.
Nutritional support has become a well established
component in patient care. Since its introduction
in the late sixties, total parenteral nutrition (TPN)
has enabled the survival of many patients and
reduced the incidence of malnutrition related to a
variety of digestive and non-digestive diseases.
Primary immunodeficiencies (PID) consist in approximately
80 distinct diseases that disturb development
and/or function of the immune system.
Lysosomal storage diseases, representing to about
forty distinct genetic entities, mostly autosomal
recessive, are due mainly to abnormalities of the
catabolism of macromolecules: glycolipids, glycoproteins,
The current knowledge in molecular biology has led for many years now to a concept that has focused
on the goal of treating genetic disorders by influencing the genetic make-up of cells, organs and body
systems such as the immune system. Widely performed research in vitro and in animals has progressed
so much that we are just at the beginning of a completely new therapeutic regimen in medicine which
has never existed before. Enough information has been gained to conclude that human gene transfer is
possible and that it can evoke biological responses that are relevant to human disease .