Use of manipulated stem cells for prenatal therapy.
Prenatal stem cell therapy has broad potential for therapeutic application. "Stem cells" of interest include multipotent adult-derived stem cells, cord blood, amniotic fluid, or fetal stem cells, and embryonic or induced pluripotent stem cells. Potential manipulations of stem cells prior to their administration may include harvest, processing, enrichment, expansion, and genetic transduction. A complete description of the methodology related to all of the above is well beyond the scope of this chapter. In the interest of practical application and proven efficacy, we limit our description to adult-derived hematopoietic stem cells (HSCs) and their application to in utero transplantation with or without HSC-targeted gene transfer.
Department of Surgery, Children's Center for Fetal Research and Center for Fetal Diagnosis and Treatment, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Jessica L
JL
Roybal
Pablo
P
Laje
Jesse D
JD
Vrecenak
Alan W
AW
Flake
D000818:Animals; D001854:Bone Marrow Cells / Q000166:cytology; D002469:Cell Separation; D005822:Genetic Vectors / Q000235:genetics; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006412:Hematopoietic Stem Cells / Q000166:cytology / Q000378:metabolism*; D007267:Injections; D016086:Lentivirus / Q000235:genetics; D051379:Mice; D011295:Prenatal Care / Q000379:methods*; D014161:Transduction, Genetic; D014817:Vitelline Membrane / Q000098:blood supply