title: Gene transfer into human haematopoietic stem cells.
abstract: This review of gene transfer to the human haematopoietic system (1) describes the different vectors used to transduce genes into stem cells, emphasizing retroviruses that have already shown their efficiency and 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 long-lasting, 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.
authors.affiliation: Institut d'Embryologie Cellulaire et Moléculaire du Collège de France et du CNRS, France.
authors.forename: B
authors.initials: B
authors.lastname: Péault
authors.forename: P
authors.initials: P
authors.lastname: Charbord
mesh_terms: D018014:Gene Transfer Techniques; D005822:Genetic Vectors; D019650:Hematopoietic Stem Cell Mobilization; D006412:Hematopoietic Stem Cells; D006801:Humans