Targeted retroviral transduction of c-kit+ hematopoietic cells using novel ligand display technology
Targeted retroviral transduction of c-kit+ hematopoietic cells using novel ligand display technology
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DOI：
10.1182/blood-2003-10-3717
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发表时间：
2004-11-01
期刊：
影响因子：
20.3
通讯作者：
Casimir, C
中科院分区：
文献类型：
--
作者：
Chandrashekran, A;Gordon, MY;Casimir, C
通过开发能够靶向将基因递送至特定细胞群体的载体，将极大地增强对多种疾病的基因治疗。我们在这里描述了一种新的靶向策略的基础上，利用逆转录病毒的能力，将宿主细胞蛋白纳入病毒颗粒的表面，因为他们芽通过质膜的高效靶向转导。亲嗜性逆转录病毒颗粒中产生的细胞工程表达膜结合形式的干细胞因子（mbSCF），以严格的c-kit（SCF受体）-依赖的方式，高效率的人细胞系和原代细胞。有效的靶向载体的可用性为使用体内基因递送的新一代疗法的开发提供了平台。(C)2004年，美国血液学会。
Gene therapy for a wide variety of disorders would be greatly enhanced by the development of vectors that could be targeted for gene delivery to specific populations of cells. We describe here high-efficiency targeted transduction based on a novel targeting strategy that exploits the ability of retroviruses to incorporate host cell proteins into the surface of the viral particle as they bud through the plasma membrane. Ecotropic retroviral particles produced in cells engineered to express the membrane-bound form of stem cell factor (mbSCF) transduce both human cell lines and primary cells with high efficiency in a strictly c-kit (SCF receptor)-dependent fashion. The availability of efficient targeted vectors provides a platform for the development of a new generation of therapies using in vivo gene delivery. (C) 2004 by The American Society of Hematology.