hemophilia B

The clinical trial of gene therapy on four hemophilia B patients

四例血友病B患者基因治疗的临床试验

Study on Gene Mutations of Factor ⅸ Gene of Hemophilia B Patients from China

27例乙型血友病患者Ⅸ因子基因突变研究

Rabbit experiments and related safety assessment study of gene transfer therapy for hemophilia B

基因转移治疗血友病B的动物试验和安全性探讨

Study on mutations in hemophilia B patient factor ⅸ gene

血友病B基因突变的研究

Clinical studies on gene therapy for hemophilia B

血友病乙基因治疗临床研究

Prenatal diagnosis of hemophilia B by factor IX gene analysis

血友病B的产前基因诊断

Progressions of gene therapy studies for hemophilia B were introduced in this paper .

就血友病B的基因治疗研究进展及所存在的问题作一综述。

Safety assessments required for animal test and preclinical trial of hemophilia B gene therapy

血友病B基因治疗临床试验的安全性研究

Objective To study the mechanism of molecular defect of factor ⅸ( F ⅸ) in hemophilia B patient .

目的为了研究血友病B因子Ⅸ（FⅨ）分子缺陷的发生机制。

The Construction and the Study of Expression Retroviral Vector of Gene Therapy for Hemophilia B by Muscle Cells

肌细胞途径血友病B基因治疗高表达载体的构建及表达研究

Some genetic diseases , such as hemophilia B , are partly caused by a given point mutation .

一些遗传性疾病，如乙型血友病，有部分是由于Ⅸ因子核苷酸序列中特定位点点突变引起的。

Studies on the Phenotype and Genetic Stability of Coagulation Factor ⅸ - deficient Mouse Model for Human Hemophilia B

凝血因子Ⅸ基因剔除小鼠遗传稳定性及其临床表型研究

Gene Therapy for Hemophilia B

血友病B的基因治疗

Establishment of a Hemophilia B Transgenic Mouse Model on the Basis of Coagulation Factor ⅸ Gene Knock-out Mouse

基于凝血因子Ⅸ基因剔除小鼠建立血友病乙转基因动物模型

DXS102 locus can be used as a promising marker for gene diagnosis in hemophilia B family .

结论DXS102座位连锁分析有望成为一种有效的血友病B基因诊断的方法。

The F ⅸ∶ Ag of the plasma in patient with severe hemophilia B were tallied with clinical diagnosis .

重症乙型血友病人血浆FⅨ∶Ag测得值与临床诊断相符。

All of 10 hemophilia B families were made final diagnosis by using 6 extragenic loci of factor ⅸ respectively .

联合FⅨ基因外6个STR位点对血友病B家系进行遗传连锁分析，使10个家系全部得到诊断。

Objective : To develop experimentally gene therapy for hemophilia B by using C2C12 cells and C3H mice as models .

目的：以小鼠C2C12成肌细胞为对象开展血友病乙基因治疗研究。

A 29 year old woman with a family history of hemophilia B asked for prenatal diagnosis in the 8th week of pregnancy .

一例有血友病B家族史的29岁孕妇，于妊娠8周时要求对胎儿进行诊断。

The bio-therapy and gene-therapy of hemophilia B in China

中国血友病B生物基因治疗研究状况

Hemophilia B is a X chromosome-linked recessive bleeding disorder which results from a deficiency in the coagulation factor IX.

血友病B是X-连锁的隐性遗传疾病，是由于凝血因子IX缺乏引起。

Hemophilia B is an X chromsome-linked recessive factor IX ( hFIX ) deficiency bleeding disorder occurring 1 in 30,000 males .

（一）血友病B是血浆凝血IX因子缺乏所导致的X连锁隐性出血性遗传病，在男性患病率约为三万分之一。

In this study , long-term , high-level expression of FIX mediated by recombinant AAV vectors in hemophilia B mice was reported . The bleeding symptom of the treated mice was significantly ameliorated .

本研究采用重组AAV为载体，使凝血因子IX基因在血友病B小鼠体内高效、长期稳定地表达，从而改善小鼠的出血症状，提高各项凝血指标，为今后的临床试验打下了基础。

The gene frequencies were 0.19.The female heterozygous showed all four bands and the frequencies were 0.31.The new polymorphism seems to be useful for carrier detection and prenatal diagnosis of hemophilia B in Chinese population .

该发现有利于我国血友病B携带者筛查和产前基因诊断。

The incidence was 2.5 / 105 , hemophilia A being the most commonly seen ( 28 / 36 ) . There were 5 cases of hemophilia B , 2 cases of C and one case of factor V deficiency .

甲型血友病最为多见（28/36饲），乙型5例，丙型2例，因子Ⅴ缺乏1例。