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Abstract |
Over the last 50 years, advances in molecular biology and genetics have made genome
engineering possible.
This resulted, amongst many applications, in the medical development of gene therapy. The
latter was first successfully used to treat rare inherited disorders of the immune system
because of specificities of the immune cells (T lymphocytes). Based on a `gene addition’
strategy that requires viruses as gene carriers, gene therapy is now being used to treat several
inherited disorders of the immune system while applications to immunotherapy of some
cancers have also been successfully implemented. The next challenge will be to edit mutated
genes to fix mutations rather than gene addition, a promising strategy that is still in its infancy. |
