因镰状细胞疾病将β球蛋白基因转移至人骨髓

β-globin gene transfer to human bone marrow for sickle cell disease
作者:Zulema Romero
期刊: J CLIN INVEST2013年1月8期123卷

Introduction

Sickle cell disease (SCD) is one of the most common monogenic disorders worldwide and is a major cause of morbidity and early mortality (1). Although SCD is well characterized, there is still no ideal long-term treatment. Current therapies are based on induction of fetal hemoglobin (HbF) to inhibit polymerization of sickle hemoglobin (HbS) (2) and cell dehydration (3) or reduction of the percentage of HbS by transfusions (4). Allogeneic HSC transplantation (HSCT) from BM or umbilical cord blood (UCB) is a potentially curative therapy, although only a small percentage of patients have undergone this procedure, mostly children with severe symptoms who had HLA-matched siblin


学科代码:其他   关键词:none
来源: J Clin Invest.
顶一下(0
您可能感兴趣的文章
    发表评论网友评论(0)
      发表评论
      登录后方可发表评论,点击此处登录