用于血红蛋白病的宫内造血细胞移植。
In utero hematopoietic cell transplantation for hemoglobinopathies.
作者信息
Derderian S Christopher, Jeanty Cerine, Walters Mark C, Vichinsky Elliott, MacKenzie Tippi C
机构信息
Department of Surgery, Eli and Edythe Broad Center of Regeneration Medicine, University of California San Francisco San Francisco, CA, USA.
Children's Hospital and Research Center Oakland Oakland, CA, USA.
出版信息
Front Pharmacol. 2015 Jan 12;5:278. doi: 10.3389/fphar.2014.00278. eCollection 2014.
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.
摘要
宫内造血细胞移植（IUHCTx）是一种很有前景的策略，可规避出生后造血干细胞（HSC）移植面临的挑战。IUHCTx的目标是在免疫成熟前将供体细胞引入未成熟宿主，从而诱导供体特异性耐受。因此，该技术有潜力避免使用细胞毒性药物进行宿主清髓预处理。在过去二十年中，人们多次尝试进行IUHCTx以治愈多种潜在的先天性异常，但成效有限。在本综述中，我们将简要回顾IUHCTx的历史，并对其临床应用的一个目标疾病——重型α地中海贫血发表看法。
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