In-Utero Gene Therapy or IUGT (Before Symptoms Appear)
There are about 400 approved clinical gene therapy protocols available world-wide, but all of them treat paediatric and adult patients after birth. However, genetic diseases can also be treated by the gene transfer in the faetus and the technique is described as 'in-utero gene therapy (IUGT)'. For a number of neurological genetic diseases (e.g. Tay-Sachs, Lesch-Nyhan, etc.), where irreversible damage may be caused during gestation before the birth, treatment may be needed before birth to allow the birth of a normal baby.
However, decision about the target tissue and the availability of technique to safely and efficiently deliver the gene at the target tissue (e.g. brain) are the major limitations for treating these diseases at the level of faetus. In view of this, diseases involving an easily accessible cell type (e.g. haematopoietic stem or progenitor cells or HSCs) will be the choice for IUGT. These diseases may improve immunodeficiency, thalassemia, osteopetrosis, etc.
