You Position: Home > Paper
子宫内胎儿基因治疗研究进展
( views:298, downloads:216 )
- Author:
- No author available
- Journal Title:
- CHINESE JOURNAL OF OBSTETRICS AND GYNECOLOGY
- Issue:
- 9
- DOI:
- 10.3760/cma.j.issn.0529-567x.2010.09.018
- Key Word:
- No keyword available
Abstract： @@ 子宫内胎儿基因治疗(宫内基因治疗)是指将正常基因导入靶细胞代替遗传缺陷的基因,或关闭、抑制异常表达的基因,以达到预防和治疗遗传性疾病的目的.随着人类产前诊断技术和分子遗传学研究的进步,许多遗传性疾病在早期妊娠就可得到诊断.若能在孕妇分娩前治疗遗传性疾病,可将疾病的危害性降到最低程度,减轻家庭和社会负担.目前,宫内移植同种基因的造血干细胞(hemopoietic stem cell,HSC)已在动物模型及人类胎儿取得成功.但由于多种原因,这种新的治疗方法仅限于有严重免疫缺陷的胎儿[1].故宫内基因治疗还处于研究阶段,有许多问题亟待解决.
- [1]Buckley SM,Acosta-Saltos AD,Elston KE,etc.Efficient gene delivery to the adult and fetal CNS using pseudotyped non-integrating lentiviral vectors.[J].2009,16(4).
- [2]Reay DP,Bilbao R,Koppanati BM,etc.Full-length dystrophin gene transfer to the mdx mouse in utero.[J].2008,15(7).
- [3]Rissanen TT,Hiltunen MO,Manninen H,etc.Angiographically guided utero-placental gene transfer in rabbits with adenoviruses, plasmid/liposomes and plasmid/polyethyleneimine complexes.[J].2001,8(10).
- [4]Xiao-Guang Chen,Huan-zhang ZHU,Ju-li GONG,etc.Efficient delivery of human clotting factor Ⅸ after injection of lentiviral vectors in utero[J].2004,(6).
- [5]Shang D,Niiya M,Endo M.Correction of ADAMTS13 deficiency by in utero gene transfer of lentiviral vector encoding ADAMTS13 genes.[J].2009,17(1).
- [6]Ye, L,Sun, XF,Chang, JC,etc.Induced pluripotent stem cells offer new approach to therapy in thalassemia and sickle cell anemia and option in prenatal diagnosis in genetic diseases[J].2009,106(24).
- [7]Waddington SN,Kennea NL,Buckley SM,etc.Fetal and neonatal gene therapy: benefits and pitfalls.[J].2004,11(Suppl.1).
- [8]Koppanati BM,Li J,Reay DP,etc.Improvement of the mdx mouse dystrophic phenotype by systemic in utero AAV8 delivery of a minidystrophin gene.[J].2010,17(11).
- [9]Penn MS,Mangi AA.Genetic enhancement of stem cell engraftment, survival, and efficacy.[J].2008,102(12).
- [10]Schoeberlein A,Wagner A,Surbek D.Perinatal stem-cell and gene therapy for hemoglobinopathies.[J].2008,13(4).
- [11]MacVinish LJ,Jezzard S,Cuthbert AW,etc.Intra-amniotic delivery of CFTR-expressing adenovirus does not reverse cystic fibrosis phenotype in inbred CFTR-knockout mice.[J].2008,16(5).
- [12]Santore MT,Roybal JL,Flake AW.Stem cell and genetic therapies for the fetus.[J].2010,15(1).
- [13]Waddington SN,Kramer MG,Hernandez Alcoceba R,etc.In Utero gene therapy: current challenges and perspectives.[J].2005,11(5).
- [14]Merianos DJ,Tiblad E,Santore MT.Maternal alloantibodies induce a postnatal immune response that limits engraftment following in utero hematopoietic cell transplantation in mice.[J].2009,119(9).
- [15]Kanzaki T,Endoh M,Morishita R,etc.Fetal gene transfer by intrauterine injection with microbubble-enhanced ultrasound.[J].2002,5(5).
- [16]Porada CD,Park PJ,Almeida Porada G,etc.Gestational age of recipient determines pattern and level of transgene expression following in utero retroviral gene transfer.[J].2005,11(2).
- [17]Schoeberlein A,Wagner AM,Surbek D.Fetal gene therapy: opportunities and risks.[J].2009,61(10).