M. Yu et al., INTRACELLULAR IMMUNIZATION OF HUMAN FETAL CORD-BLOOD STEM PROGENITOR CELLS WITH A RIBOZYME AGAINST HUMAN-IMMUNODEFICIENCY-VIRUS TYPE-1, Proceedings of the National Academy of Sciences of the United Statesof America, 92(3), 1995, pp. 699-703
Successful treatment of human immunodeficiency virus infection may ult
imately require targeting of hematopoietic stem cells. Here we used re
troviral vectors carrying the ribozyme gene to transduce CD34(+) cells
from human fetal cord blood. Transduction and ribozyme expression had
no apparent adverse effect on cell differentiation and/or proliferati
on. The macrophage-like cells, differentiated from the stem/progenitor
cells in vitro, expressed the ribozyme gene and resisted infection by
a macrophage tropic human immunodeficiency virus type 1. These result
s suggest the feasibility of stem cell gene therapy for human immunode
ficiency virus-infected patients.