Boggs S S
Department of Radiation Oncology, University of Pittsburgh School of Medicine, Pennsylvania.
Int J Cell Cloning. 1990 Mar;8(2):80-96. doi: 10.1002/stem.5530080202.
Ideally, gene therapy would correct the specific gene defect without adding potentially harmful extraneous DNA sequences. Such correction can be obtained with homologous recombination between input DNA sequences and identical (homologous) sequences in the genomic target gene. The development of techniques for obtaining virtually pure populations of hematopoietic stem cells should permit the use of the highly efficient nuclear microinjection methods for transfer of DNA. These techniques combined with new highly sensitive methods for detecting cells with the specified genetic modification of nonexpressed genes would make homologous recombination-mediated gene therapy feasible for hematopoietic stem cells. These advances are reviewed with particular emphasis on approaches to targeted gene modification of hematopoietic stem cells and speculation on directions for future research.
理想情况下，基因治疗应能纠正特定的基因缺陷，而不添加潜在有害的外来DNA序列。通过输入DNA序列与基因组靶基因中相同（同源）序列之间的同源重组，可以实现这种纠正。获取几乎纯的造血干细胞群体技术的发展，应能允许使用高效的核显微注射方法来转移DNA。这些技术与用于检测具有未表达基因特定基因修饰细胞的新型高灵敏度方法相结合，将使同源重组介导的基因治疗对造血干细胞成为可行。本文对这些进展进行了综述，特别强调了造血干细胞靶向基因修饰的方法以及对未来研究方向的推测。
