Gene marking.
Gene marking studies were the first gene transfer protocols to enter clinical practice. To date, clinical marking studies have been limited to the hematopoietic stem cell and its progeny. In this setting, they have provided valuable information about stem cell biology, the factors that influence gene transfer efficiency, and the mechanism of relapse in patients receiving stem cell rescue as therapy for malignant disease. Second-generation studies are beginning to provide even more information about a wider variety of clinical and biological issues. Although marker studies have been useful, it is becoming apparent that the indicator genes used up to now have a number of undesirable characteristics. Future applications of marking, in the hematopoietic system and elsewhere, will require the use of marker elements that will not produce any modification of the cells' behavior. Finally, marker studies have proved safe so far, but follow-up of the treated patients continues.
Brenner
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Division of Bone Marrow Transplantation, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.
D000818:Animals; D016026:Bone Marrow Transplantation; D017690:Cell Transplantation; D003257:Consumer Product Safety; D005544:Forecasting; D018014:Gene Transfer Techniques; D017930:Genes, Reporter; D005822:Genetic Vectors; D006412:Hematopoietic Stem Cells; D006801:Humans; D014184:Transplantation, Homologous