Transduction of Human CD34 + Cells That Mediate Long-Term Engraftment of NOD/SCID Mice by HIV Vectors

作者：Hiroyuki Miyoshi, Kent A. Smith, Donald E. Mosier, Inder M. Verma, Bruce E. Torbett · 发表于：Science · 年份：1999 · DOI：10.1126/science.283.5402.682 · 被引用次数：636 · 研究领域：Virus-based gene therapy research、RNA Interference and Gene Delivery、CRISPR and Genetic Engineering

Efficient gene transfer into human hematopoietic stem cells (HSCs) is an important goal in the study of the hematopoietic system as well as for gene therapy of hematopoietic disorders. A lentiviral vector based on the human immunodeficiency virus (HIV) was able to transduce human CD34+ cells capable of stable, long-term reconstitution of nonobese diabetic/severe combined immunodeficient (NOD/SCID) mice. High-efficiency transduction occurred in the absence of cytokine stimulation and resulted in transgene expression in multiple lineages of human hematopoietic cells for up to 22 weeks after transplantation.