Mozzetta Chiara, Minetti Giulia, Puri Pier Lorenzo
Dulbecco Telethon Institute at Fondazione Santa Lucia/EBRI, Via di Fosso Fiorano, 64-00143 Roma, Italy.
Int J Biochem Cell Biol. 2009 Apr;41(4):701-10. doi: 10.1016/j.biocel.2008.08.033. Epub 2008 Sep 2.
Current evidence supports the therapeutic potential of pharmacological interventions that counter the progression of genetic disorders by promoting regeneration of the affected organs or tissues. The rationale behind this concept lies on the evidence that targeting key events downstream of the genetic defect can compensate, at least partially, the pathological consequence of the related disease. In this regard, the beneficial effect exerted on animal models of muscular dystrophy by pharmacological strategies that enhance muscle regeneration provides an interesting paradigm. In this review, we describe and discuss the potential targets of pharmacological strategies that promote regeneration of dystrophic muscles and alleviate the consequence of the primary genetic defect. Regenerative pharmacology provides an immediate and suitable therapeutic opportunity to slow down the decline of muscles in the present generation of dystrophic patients, with the perspective to hold them in conditions such that they could benefit of future, more definitive, therapies.
目前的证据支持药物干预的治疗潜力，这些干预通过促进受影响器官或组织的再生来对抗遗传疾病的进展。这一概念背后的基本原理基于这样的证据，即针对基因缺陷下游的关键事件可以至少部分地补偿相关疾病的病理后果。在这方面，通过增强肌肉再生的药理策略对肌肉营养不良动物模型产生的有益作用提供了一个有趣的范例。在这篇综述中，我们描述并讨论了促进营养不良性肌肉再生并减轻原发性基因缺陷后果的药理策略的潜在靶点。再生药理学为减缓当代营养不良患者肌肉衰退提供了一个直接且合适的治疗机会，以期使他们处于能够受益于未来更明确疗法的状态。
