In utero hematopoietic cell transplantation for hemoglobinopathies

S Christopher Derderian, Cerine Jeanty, Mark C Walters, Elliott Vichinsky, Tippi C MacKenzie, S Christopher Derderian, Cerine Jeanty, Mark C Walters, Elliott Vichinsky, Tippi C MacKenzie

Abstract

In utero hematopoietic cell transplantation (IUHCTx) is a promising strategy to circumvent the challenges of postnatal hematopoietic stem cell (HSC) transplantation. The goal of IUHCTx is to introduce donor cells into a naïve host prior to immune maturation, thereby inducing donor-specific tolerance. Thus, this technique has the potential of avoiding host myeloablative conditioning with cytotoxic agents. Over the past two decades, several attempts at IUHCTx have been made to cure numerous underlying congenital anomalies with limited success. In this review, we will briefly review the history of IUHCTx and give a perspective on alpha thalassemia major, one target disease for its clinical application.

Keywords: alpha thalassemia; chimerism; fetal therapy; in utero transplantation; tolerance.

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