In utero stem cell transplantation and gene therapy: Recent progress and the potential for clinical application.
Advances in prenatal diagnosis have led to the prenatal management and treatment of a variety of congenital diseases. Although surgical treatment has been successfully applied to specific anatomic defects that place the fetus at a risk of death or life-long disability, the indications for fetal surgical intervention have remained relatively limited. By contrast, prenatal stem cell and gene therapy await clinical application, but they have tremendous potential to treat a broad range of genetic disorders. If there are biological advantages unique to fetal development that favor fetal stem cell or gene therapy over postnatal treatment, prenatal therapy may become the preferred approach to the treatment of any disease that can be prenatally diagnosed and cured by stem cell or gene therapy. Here, we review the field including recent progress toward clinical application and imminent clinical trials for cellular and gene therapy.
Children's Center for Fetal Research, Children's Hospital of Philadelphia, University of Pennsylvania School of Medicine, Philadelphia, PA, USA.
Lauren E
LE
McClain
Children's Center for Fetal Research, Children's Hospital of Philadelphia, University of Pennsylvania School of Medicine, Philadelphia, PA, USA. Electronic address: flake@email.chop.edu.
Alan W
AW
Flake
D005260:Female; D005315:Fetal Diseases / Q000175:diagnosis / Q000628:therapy*; D046128:Fetal Therapies; D015316:Genetic Therapy; D005822:Genetic Vectors; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D045164:Mesenchymal Stem Cell Transplantation; D011247:Pregnancy; D011296:Prenatal Diagnosis; D033581:Stem Cell Transplantation
gene therapy; hematopoietic stem cell transplantation; in utero; prenatal therapy