Can gene therapy be used as treatment for down syndrome?
No. Gene therapy alters a person’s genetic code, generally by inserting a genetic sequence. Trisomy 21 (downs syndrome) is caused by the presence of a copy (or part of a copy) of the 21st chromosome. Genetic therapy would not be able to remove this extra chromosome. It might be possible (in the future) to “turn off” the extra chromosome, and stop trisomy-21 at the embryo stage. However, by the time a person is born, the physical damage from downs syndrome has been done. After a few months to a year or so, the mental damage from downs syndrome will also be done. So no “treatment” for downs syndrome will be possible using genetic modification such as gene therapy.
