Expression of human adenosine deaminase in nonhuman primates after retrovirus-mediated gene transfer.

作者：Philip W. Kantoff, Alfred P. Gillio, Jeanne R. McLachlin, Claudio Bordignon, Martin A. Eglitis, Nancy A. Kernan, R C Moen, Donald B. Kohn, S F Yu, Evelyn M. Karson · 发表于：The Journal of Experimental Medicine · 年份：1987 · DOI：10.1084/jem.166.1.219 · 被引用次数：183 · 研究领域：Virus-based gene therapy research、RNA Interference and Gene Delivery、CRISPR and Genetic Engineering

Primate bone marrow cells were infected with a retroviral vector carrying the genes for human adenosine deaminase (h-ADA) and bacterial neomycin resistance (neor). The infected cells were infused back into the lethally irradiated donor animals. Several monkeys fully reconstituted and were shown to express the h-ADA and neor genes at low levels in their recirculating hematopoietic cells for short periods of time.