IUGT (in utero gene transfer)

Used to correct genetic defects of somatic cells by transfer of gene(s) into the unborn fetus. Its advantage is that it can be applied early during embryonic development before irreversible damage occurs, e.g., in various neurological diseases (Tay-Sachs, Niemann-Pick, Lesh-Nyhan, Sandhoff, Leigh, leukodystrophies, gangliosidoses, immunological disorders, thalassemias, osteopetrosis). It is less likely that IUGT involves vector or cell rejection. The treatment of hematopoietic stem cells may be more successful. Animal experiments have yielded encouraging results. Its drawbacks are the potential risk to the mother and the fetus. Also, the vector DNA may cause undesirable insertions or mutations in the germline. ART, gene therapy, Heikkila A et al 2001 Gene Ther 8:784.