[Recent advances in gene therapy and future prospects].
Gene therapy, which is treatment of diseases by introducing normal genes into the body, is becoming feasible as the result of advances in genetic engineering. The hematopoietic stem cells have been considered as the appropriate target for gene transfer in many genetic diseases for which allogeneic bone marrow transplantation has been employed successfully. However, there are still many problems to be solved. In particular, expression from retrovirally transduced genes in bone marrow cells has been transient and unstable. On the other hand, an alternative approach to somatic cell gene therapy using nonhematopoietic cells, including skin fibroblasts, endothelial cells, keratinocytes, and lymphocytes, has been shown to possess several advantages. This kind of approach is usually applied to supplementation therapy in not only hereditary disorders but also various acquired diseases, such as cancer or infectious diseases. Recently, clinical application of gene transfer into lymphocytes to treat cancer and immunodeficiency have been approved at NIH (USA). The trial could represent the start of a new era in molecular medicine.
Ozawa
K
K
Department of Hematology-Oncology, University of Tokyo, Japan.
D016026:Bone Marrow Transplantation; D015316:Genetic Therapy / Q000639:trends*; D006412:Hematopoietic Stem Cells; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000628:therapy*; D009369:Neoplasms / Q000628:therapy*; D014161:Transduction, Genetic; D014162:Transfection