Liu Li, Parekh-Olmedo Hetal, Kmiec Eric B
Department of Biological Sciences, University of Delaware, Newark, Delaware 19716, USA.
Nat Rev Genet. 2003 Sep;4(9):679-89. doi: 10.1038/nrg1156.
A technique that can direct the repair of a genetic mutation in a human chromosome using the DNA repair machinery of the cell is under development. Although this approach is not as mature as other forms of gene therapy and fundamental problems continue to arise, it promises to be the ultimate therapy for many inherited disorders. There is a continuing effort to understand the potential and the limitations of this controversial approach.
一种利用细胞的DNA修复机制来指导人类染色体基因突变修复的技术正在研发中。尽管这种方法不如其他形式的基因疗法成熟,且基本问题仍不断出现,但它有望成为许多遗传性疾病的终极疗法。人们一直在努力了解这种有争议方法的潜力和局限性。