Bretag Allan H
Sansom Institute, School of Pharmacy and Medical Sciences, University of South Australia, Adelaide, South Australia 5000, Australia.
Nature. 2007 Dec 20;450(7173):E23; discussion E23-5. doi: 10.1038/nature06437.
Human muscular dystrophies are devastating and incurable inherited diseases. Hopes of progress towards therapy of muscular dystrophies were aroused when Sampaolesi et al. reported "extensive recovery of dystrophin expression, normal muscle...function", and "remarkable clinical amelioration" in golden retriever muscular dystrophy dogs treated with 'mesoangioblasts'. Here I re-examine their results, showing how their assessments might be flawed and their conclusions overstated. Further studies will be required to evaluate fully the clinical potential of this work.
人类肌肉萎缩症是毁灭性的、无法治愈的遗传性疾病。当桑帕奥莱西等人报告在用“中胚层血管母细胞”治疗的金毛猎犬型肌肉萎缩症犬中出现“抗肌萎缩蛋白表达广泛恢复、肌肉……功能正常”以及“临床症状显著改善”时，人们燃起了在肌肉萎缩症治疗方面取得进展的希望。在此，我重新审视他们的研究结果，展示其评估可能存在的缺陷以及结论可能被夸大的情况。要全面评估这项研究工作的临床潜力，还需要进一步的研究。
