In utero therapy for congenital disorders using amniotic fluid stem cells

摘要: Congenital diseases are responsible for over a third of all pediatric hospital admissions. Advances in prenatal screening and molecular diagnosis have allowed the detection many life-threatening genetic early gestation. In utero transplantation (IUT) with stem cells could cure affected fetuses but so far humans, successful IUT using allogeneic hematopoietic (HSCs), has been limited to severe immunologic defects more recently mesenchymal cell transplantation, improved phenotype osteogenesis imperfecta. The options preemptive treatment congenital by or gene therapy changes perspective since it may avoid need postnatal reduce future costs. Amniotic fluid (AFS) isolated characterized human, mice, rodents, rabbit, sheep potential source therapeutic applications disorders prenatally postnatally. Gene transfer long-term transgenic protein expression is feasible. Recently, pre-clinical autologous transduced achieved fetal minimally invasive ultrasound guided injection techniques. Clinically relevant levels were expressed blood transplanted lambs at least 6 months. also demonstrated repair range disease models such as neurological disorders, tracheal repair, bladder injury, diaphragmatic hernia neonates adults. These results encouraging, bring personalized tissue engineering closer clinic.