Université Paris Diderot, Sorbonne Paris Cité, Hôpital St. Louis 1, Avenue Claude Vellefaux, 75475 Paris cedex 10, France.
Mol Oncol. 2013 Apr;7(2):224-31. doi: 10.1016/j.molonc.2013.01.006. Epub 2013 Feb 11.
The relative genetic simplicity of leukaemias, the development of which likely relies on a limited number of initiating events has made them ideal for disease modelling, particularly in the mouse. Animal models provide incomparable insights into the mechanisms of leukaemia development and allow exploration of the molecular pillars of disease maintenance, an aspect often biased in cell lines or ex vivo systems. Several of these models, which faithfully recapitulate the characteristics of the human disease, have been used for pre-clinical purposes and have been instrumental in predicting therapy response in patients. We plea for a wider use of genetically defined animal models in the design of clinical trials, with a particular focus on reassessment of existing cancer or non-cancer drugs, alone or in combination.
白血病的相对遗传简单性，其发展可能依赖于有限数量的起始事件，这使得它们成为疾病建模的理想选择，尤其是在小鼠中。动物模型为白血病发展机制提供了无与伦比的见解，并允许探索疾病维持的分子支柱，这在细胞系或体外系统中往往存在偏差。其中一些模型忠实地再现了人类疾病的特征，已被用于临床前目的，并有助于预测患者的治疗反应。我们呼吁在临床试验设计中更广泛地使用基因定义明确的动物模型，特别关注单独或联合使用现有癌症或非癌症药物的重新评估。
