[Gene therapy for monogenic inherited diseases].

BACKGROUND

Monogenic inherited disorders are caused by a mutation in one single gene. Each of these disorders is very rare, but as there are many thousand different monogenic disorders, they represent a significant health problem. Most monogenic disorders are severe and have no cure. Gene therapy will therefore often be the only possible treatment.

MATERIAL AND METHODS

Characterisation of the gene is necessary for the development of gene therapy, and at the present time only a limited number of the genes in relation to the total number of the severe monogenic disorders is known.

RESULTS

Clinical trials for some of the monogenic disorders, such as cystic fibrosis, have been going on for many years.

INTERPRETATION

In spite of the tremendous effort, it is so far not documented that patients have been cured of a monogenic disorder by gene therapy.