干细胞宫内基因治疗的研究进展

Progress in research on in utero gene therapy with hemotopoietic stem cells transplantation

遗传病学、分子生物学研究的深入以及产前诊断技术的发展和应用,为妊娠早期诊断胎儿遗传性疾病创造了重要的条件,宫内基因治疗成为妊娠早期预防和治疗该类疾病的有效途径。由于干细胞具有多能性或全能性、自我更新能力和高度增殖能力等特性,使得其在宫内基因治疗方面具有非常重要的理论研究意义和临床应用价值。该文就目前国内、外对干细胞应用于宫内基因治疗的最新研究成果及有待解决的问题作以综述。

The deepening in genetic and molecular biological researches and development and application of prenatal diagnosis techniques have created important conditions for diagnosis of fetal genetic diseases in the first trimester of pregnancy. Therefore, in utero gene therapy has become an effective method for preventing and treating such diseases in early pregnancy. Because of its characteristics such as pluripotency or totipotency, self-renewal and highly proliferous abilities, in utero gene therapy with stem cell transplantation is of very important theoretical research significance and clinical application values. This paper reviewed the latest research results and problems to be solved in this field.