In Utero Hematopoietic Cell Transplantation for Hemoglobinopathies

Overview

Journal Front Pharmacol

Date 2015 Jan 29

PMID 25628564

Citations 10

Authors

S Christopher Derderian

Cerine Jeanty

Mark C Walters

Elliott Vichinsky

Tippi C MacKenzie

Affiliations

Soon will be listed here.

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.

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