HIGH EFFICIENCY EX VIVO TRANSDUCTION OF HEMATOPOIETIC STEM CELLS BY RECOMBINANT RETROVIRAL PREPARATIONS

摘要

Compositions and methods for the efficient ex vivo introduction of nucleic acid into hematopoietic stem cells mediated by recombinant retrovirus particles is described. The recombinant vector constructs carried by the recombinant retrovirus particles code for the production of a desired gene product having a therapeutic application from a gene of interest. Upon re-introduction into a patient, the transduced hematopoietic stem cells produce a desired gene product in an amount sufficient to treat a particular disease state.