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Updated: Oct 9, 2026

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
Published on: November 4, 2018
[Gene therapy for monogenic inherited diseases]
1Avdeling for medisinsk genetikk Rikshospitalet 0027 Oslo. k.h.orstavik@ioks.uio.no
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.
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