Gene Therapy for Hemoglobinopathies The State of the Field and the Future

被引：45

作者：

Chandrakasan, Shanmuganathan ^{[1
]}

Malik, Punam ^{[2
,3
]}

机构：

[1] Cincinnati Childrens Hosp Med Ctr, CBDI, Div Hematol Oncol & Bone Marrow Transplant, Cincinnati, OH 45229 USA

[2] Cincinnati Childrens Res Fdn, Cincinnati Childrens Hosp Med Ctr, CBDI, Div Expt Hematol Canc Biol, Cincinnati, OH 45229 USA

[3] Cincinnati Childrens Res Fdn, Cincinnati Childrens Hosp Med Ctr, CBDI, Div Hematol, Cincinnati, OH 45229 USA

来源：

HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA | 2014年 / 28卷 / 02期

关键词：

Hemoglobinopathy; Thalassemia; Sickle cell disease; Gene therapy; BETA-GLOBIN GENE; SICKLE-CELL-DISEASE; BONE-MARROW-TRANSPLANTATION; LOCUS-CONTROL REGION; PLURIPOTENT STEM-CELLS; HIGH-LEVEL EXPRESSION; GAMMA-GLOBIN; THALASSEMIA-INTERMEDIA; PHENOTYPIC CORRECTION; LENTIVIRAL VECTORS;

D O I：

10.1016/j.hoc.2013.12.003

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

After nearly two decades of struggle, gene therapy for hemoglobinopathies using vectors carrying beta or gamma-globin gene has finally reached the clinical doorsteps. This was made possible by advances made in our understanding of critical regulatory elements required for high level of globin gene expression and improved gene transfer vectors and methodologies. Development of gene editing technologies and reprogramming somatic cells for regenerative medicine holds the promise of genetic correction of hemoglobinopathies in the future. This article will review the state of the field and the upcoming technologies that will allow genetic therapeutic correction of hennoglobinopathies.

引用

页码：199 / +

页数：19

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