Gene transfer into human haematopoietic stem cells

This review of gene transfer to the human haematopoietic system (1) describes the different vectors used to transduce genes into stem emphasizing retroviruses that already shown their efficiency innocuousness; (2) analyses which human cells should be targeted to ensure long-lasting engraftment; (3) indicates the different means of infecting these targets ex vivo, underscoring the role of cytokines and stromal cells; (4) recollects the methods used to evaluate transduction efficiency; and (5) gathers the results of clinical trials recently performed using human stem cells. The major conclusions are that good practice can ensure safe gene delivery to human beings and that longlasting, multilineal precursors can be transduced using retroviral vectors of marker genes or genes of therapeutic interest. However, transduction rates appear to remain relatively low, which should stimulate ongoing research on both vector design and means of ex vivo gene transfer. (C) 1997 Elsevier Science Ltd.