Gene-marking studies of hematopoietic cells

Gene-marking studies were the first approved clinical protocols introducing exogenous genetic material into human cells. Such studies were never inten ded to provide direct therapeutic benefit. Instead, they were expected to p rovide information about normal cell biology and disease pathogenesis that could not be obtained in any other way. However. the information gained fro m such studies has had a significant impact on disease management. Gene-mar king studies have provided valuable insights into the biology of the human stem cell, factors that influence the efficiency of gene transfer, mechanis ms of relapse after stem cell transplantation, and the pharmacodynamics of adoptive cellular immunotherapy. With continuing advances in gene-marking t echnology, the value of the: information provided by these studies increase s, thereby ensuring their continued relevance to the field of gene transfer . (C) 2001 The Japanese Society of Hematology.