Parekh-Olmedo H, Ferrara L, Brachman E, Kmiec E B
Department of Biological Sciences, Delaware Biotechnology Institute, University of Delaware, Newark, DE 19716, USA.
Gene Ther. 2005 Apr;12(8):639-46. doi: 10.1038/sj.gt.3302511.
The capacity to correct a mutant gene within the context of the chromosome holds great promise as a therapy for inherited disorders but fulfilling this promise has proven to be challenging. However, steady progress is being made and the development of gene repair as a viable and robust approach is underway. Here, we present some of the recent advances that are helping to shape our thinking about the feasibility and the limitations of this technique. For the most part, these advances center on understanding the regulation of the reaction and validating its application in animal models.
在染色体背景下纠正突变基因的能力作为一种治疗遗传性疾病的方法具有巨大潜力，但事实证明实现这一潜力具有挑战性。然而，正在取得稳步进展，基因修复作为一种可行且强大的方法正在发展中。在此，我们介绍一些最近的进展，这些进展有助于塑造我们对该技术的可行性和局限性的认识。在很大程度上，这些进展集中在理解反应的调控以及在动物模型中验证其应用。
