Current Strategy of Fetal Therapy I: Principles of In-utero Treatment, Pharmacologic Intervention, Stem Cell Transplantation and Gene Therapy

Abstract

AbstractFetal conditions with high morbidity are amenable for prenatal intervention. It is important that the selective and investigative nature of most procedures needs to be clarified with the family during counseling session. Fetal therapy is fostered by accurate prenatal diagnosis with advanced fetal imaging, and molecular genetics technology. The treatments can be categorized into medical treatment, stem cell transplantation and gene therapy, minimally invasive intervention, endoscopic surgery, and open hysterotomy approach. Scientific validation of their genuine benefits has been a subject of ongoing researches. Prenatal administrations of pharmaceutical agents, for prophylactic or therapeutic purposes, have been broadly adopted. Transplacental administration of betamethasone to enhance the function of pneumocytes type II in premature fetus has been widely practiced for decades, and it might be the most common ‘fetal therapy’ being performed. However, the optimal dosage and interval of prenatal steroids administration was validated only recently. More invasive route of fetal administration, such as transamniotic, direct intramuscular, and intravenous injection, may be required for other pharmacologic agents. In this article, the authors selected to review common fetal conditions whose proposed prenatal pharmacologic treatments have undergone scientific validations. In-utero stem cell transplantation and gene therapy remain highly experimental. Informed choice and clinical experiment need to be balanced when prenatal treatment is offered.