Engelsk titel: Gene therapy for monogenic inherited diseases
Läs online
Författare:
Örstavik KH
;
Apold J
Språk: Nor
Antal referenser: 9
Dokumenttyp:
Artikel
UI-nummer: 01022895
Sammanfattning
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.