向心脏导入基因：一切关于递送。
Introducing Genes to the Heart: All About Delivery.
机构信息
From the Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York.
出版信息
Circ Res. 2017 Jan 6;120(1):33-35. doi: 10.1161/CIRCRESAHA.116.310039.
Abstract
Recent clinical gene therapy trials for the treatment of heart failure have failed to meet primary efficacy endpoints and have tampered enthusiasm for the future application of cardiac gene therapy. These results have brought to light the difficulty of efficiently introducing genes into the human heart and have focused on potential problems that need to be addressed before further clinical applications. These trials however have established the safety of gene delivery vectors for cardiac targeting in humans. The sinusoidal trajectory of gene therapy continues and despite these setbacks the future of the field is promising.
摘要
最近心力衰竭的临床基因治疗试验未能达到主要疗效终点，也削弱了人们对心脏基因治疗未来应用的热情。这些结果揭示了将基因有效导入人心的困难，并强调了在进一步临床应用之前需要解决的潜在问题。然而，这些试验已经证实了心脏靶向基因传递载体在人体中的安全性。基因治疗的轨迹仍在继续，尽管存在这些挫折，该领域的未来仍然充满希望。
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