Ozawa K
Department of Hematology-Oncology, University of Tokyo, Japan.
Hum Cell. 1991 Mar;4(1):13-7.
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.
基因治疗是通过将正常基因导入体内来治疗疾病，随着基因工程的进展，它正变得可行。造血干细胞已被视为许多遗传性疾病基因转移的合适靶点，而异基因骨髓移植已在这些疾病中成功应用。然而，仍有许多问题有待解决。特别是，逆转录病毒转导基因在骨髓细胞中的表达一直是短暂且不稳定的。另一方面，使用非造血细胞（包括皮肤成纤维细胞、内皮细胞、角质形成细胞和淋巴细胞）进行体细胞基因治疗的替代方法已显示出若干优势。这种方法通常不仅应用于遗传性疾病的补充治疗，还应用于各种获得性疾病，如癌症或传染病。最近，美国国立卫生研究院（NIH）已批准将基因转移到淋巴细胞中用于治疗癌症和免疫缺陷的临床试验。该试验可能代表着分子医学新时代的开端。
