Pandolfi P P
Department of Human Genetics, Memorial Sloan-Kettering Cancer Center, Graduate School of Medical Sciences, Cornell University, New York, NY 10021, USA.
Semin Hematol. 1998 Apr;35(2):136-48.
Mouse genetics and engineered mice have become invaluable tools for the study of normal and aberrant hemopoiesis, in modeling hemopoietic disorders, and developing new therapeutic approaches using these models. However, the technological exploitation of these powerful genetic tools, as well as the scientific exploitation of the mutants which are generated is still in a primordial phase. For instance, the possibility to introduce specific point mutation, in vivo, in any desired locus, will allow the production of a second generation of mutants, which will be much more informative in the dissection of the function of any molecule. Furthermore, the possibility to interbreed mutants thus generating organisms harboring concomitantly "n" mutations in "n" loci will allow the possibility to test and validate, in vivo, the role of individual biochemical and cellular pathways.
小鼠遗传学和基因工程小鼠已成为研究正常和异常造血、模拟造血系统疾病以及利用这些模型开发新治疗方法的宝贵工具。然而，这些强大的遗传工具在技术层面的应用，以及对所产生突变体的科学利用仍处于初始阶段。例如，在体内任何所需位点引入特定点突变的可能性，将使得第二代突变体的产生成为可能，这对于剖析任何分子的功能将更具参考价值。此外，使突变体杂交从而产生在“n”个位点同时携带“n”个突变的生物体的可能性，将使得在体内测试和验证单个生化和细胞途径的作用成为可能。
