According to an article in “The Volkskrant”, in the future, it may become possible to cure unborn babies in the womb from certain genetic diseases. This may become possible by using gene therapy. Right now, the research project is still in an early stage: genetic therapy in the womb only has been tested on mouses. However, the results of these tests show positive prospects, because the American researchers that executed the tests have successfully cured the unborn mouses of the genetic disease ‘tyrosinemia type 1’. This is a rear metabolism disease. In the Netherlands, it is generally once a year that a child is born with ‘tyrosinemia type 1’.
When the baby mouses were born, it turned out the gene therapy in the womb had worked better on them then the medicine ‘nitisinone’, which is used on living patients, normally would: the test mouses stayed alive after their birth and gained weight in a better way than the mouses that were on ‘nitisinone’. The medicine ‘nitisinone’ prevents damage to the liver or – worst case scenario – death.
Even though genetic diseases like ‘tyrosinemia type 1’ do not occur very often, it would be great if we would be able to bring this occurrence down from one to none a year. This thus can – in theory – be achieved by using genetic therapy in the womb. For now, the big problem is, however, that the tests were executed on mouses. And, according to professor Christine de Die, the step from mouses to humans is huge.
Do you think executing tests on mouses is ethically sound? Or do you think there is a better way? Leave a comment and let us know!