Cellular therapy.
From a clinical perspective, prenatal transplantation has tremendous potential to broaden the current indications for reconstitution therapy and to offer a safe, efficacious, and cost-effective alternative to conventional postnatal BMT for many congenital hematopoietic diseases. Experimental work and limited clinical experience offer hope for the future. The primary experimental challenges will be to manipulate the biology of in utero HSC transplantation so that the approach will be safe and broadly applicable. This will require strategies to improve engraftment; use alternative sources of cells safely and effectively; and develop techniques for procurement, ex vivo expansion, and tissue banking of safe donor cells. The clinical challenges in the future will be to identify recipients most likely to benefit from the approach, and to define further the clinical and ethical guidelines for its application.
Flake
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Children's Hospital of Philadelphia, Pennsylvania, USA.
Zanjani
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D005260:Female; D005315:Fetal Diseases / Q000628:therapy*; D005865:Gestational Age; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D008297:Male; D011247:Pregnancy; D011261:Pregnancy Trimester, First; D011295:Prenatal Care / Q000379:methods*; D016511:Severe Combined Immunodeficiency / Q000276:immunology / Q000628:therapy*