In hereditary genetic diseases, the possibility of using gene therapy before birth has opened a new possibility for treating these serious diseases. Now, a study conducted in mice has just been published in Nature Medicine (read HERE), in which treatment was applied to mice with a serious degenerative disease, Gaucher’s disease.
Aim, methodology and results
The aim of the study was to try to prevent the neurological damage that starts before birth from developing, by inserting a healthy gene instead of the faulty one that exists in this disease. The study showed that life expectancy in the mice with Gaucher’s disease was significantly prolonged after gene therapy. Despite this, the authors say that the possible application of the findings in humans will still require complementary experiments that will undoubtedly delay their clinical use by several years.
See HERE more about current gene therapy development