Loukogeorgakis Stavros P, Flake Alan W
Surgery Unit, University College London Institute of Child Health, London, United Kingdom.
The Children's Hospital of Philadelphia, and The University of Pennsylvania School of Medicine - The Center for Fetal Diagnosis and Treatment, Philadelphia, Pennsylvania, United States.
Eur J Pediatr Surg. 2014 Jun;24(3):237-45. doi: 10.1055/s-0034-1382260. Epub 2014 Jun 19.
Advances in prenatal diagnosis have led to the development of fetal therapies for congenital disorders. Although in utero surgical intervention has been used successfully for correction of anatomical defects that cause fetal demise or long-term disability, its clinical indications remain limited. In contrast, prenatal stem cell and gene therapy might have tremendous potential to treat multiple inherited disorders, and could dramatically expand the use of fetal intervention to a wide range of anticipated pediatric and adult diseases. Despite encouraging results from studies in animal models of disease, the clinical utility of such therapies has been restricted by poor efficacy and concerns about safety. The aim of this review is to summarize experimental progress toward clinical application of in utero stem cell transplantation and gene transfer for the treatment of congenital disease.
产前诊断的进展促使了针对先天性疾病的胎儿治疗方法的发展。尽管子宫内手术干预已成功用于纠正导致胎儿死亡或长期残疾的解剖学缺陷，但其临床适应证仍然有限。相比之下，产前干细胞和基因治疗可能具有治疗多种遗传性疾病的巨大潜力，并可能大幅扩大胎儿干预在众多预期的儿科和成人疾病中的应用。尽管在疾病动物模型研究中取得了令人鼓舞的结果，但此类治疗的临床实用性受到疗效不佳和安全性担忧的限制。本综述的目的是总结子宫内干细胞移植和基因转移治疗先天性疾病临床应用的实验进展。
