[Gene transfer in human hematopoietic stem cells isolated from peripheral blood].
To insert a new genetic information by gene transfer into haemopoietic stem cells would result in expression of the transgene in progenitors and progeny of cell blood lineages. If successfull, such an approach would open interesting prospectives in the field of experimental research and in the possibility to treat genetic defects affecting blood lineages such as immune deficiencies (ADA, SCID, AIDS) or enzymes defects. Moreover progenitors could be engineered to become more resistant to chemotherapy or oncogenic process. Many parameters and technical problems are still involved in this issue, including identification, isolation and selection of the most primitive progenitors, and search for the most efficient vectors to insert new genes into the target cells. So far retroviral vectors have been shown to be the most effective but search for better vectors are still underway. Peripheral blood stem cells isolated from patients stimulated by cytokines and/or chemotherapy appear interesting target cells for genetic manipulations aimed to correct an acquired or genetic defect.
Mannoni
P
P
Centre de Thérapie génique, Institut Paoli-Calmettes, Marseille.
D018014:Gene Transfer Techniques; D005822:Genetic Vectors; D006412:Hematopoietic Stem Cells; D006801:Humans; D011446:Prospective Studies