用于治疗勃起功能障碍的基因转移疗法的首次人体试验：初步结果。
The first human trial for gene transfer therapy for the treatment of erectile dysfunction: preliminary results.
作者信息
Melman Arnold, Bar-Chama Natan, McCullough Andrew, Davies Kelvin, Christ George
机构信息
Department of Urology, Albert Einstein college of Medicine, Room 744, Forchheimer, 1300 Morris Park Avenue, Bronx, NY 10461, USA. amelman@montefiore@org
出版信息
Eur Urol. 2005 Aug;48(2):314-8. doi: 10.1016/j.eururo.2005.05.005.
OBJECTIVE
To test the safety of a single intracavernous injection of a plasmid vector (hMaxi-K) that expresses the hSlo gene, that encodes the alpha-subunit of the Maxi-K channel, for the treatment of erectile dysfunction (ED).
METHODS
Six men, thus far have fulfilled the entry criteria of the protocol and had gene transfer with hMaxi-K. Three received a dose of 500 microg and three received a dose 1000 microg of the gene product, injected intracavernously as naked DNA. Dosing at 5000 microg and higher is planned.
RESULTS
The primary end point of the phase I trial is safety. No drug-related adverse events or significant laboratory changes have occurred after the gene transfer. Moreover, there is no evidence of the gene in semen at the one copy per mug total DNA in any of the participants.
CONCLUSION
Preliminary results indicate that, in a single dose escalation study, ion channel gene transfer with hMaxi-K can be administered safely to men with ED without adverse events.
目的
测试单次海绵体内注射表达hSlo基因（该基因编码大电导钙激活钾通道α亚基）的质粒载体（hMaxi-K）治疗勃起功能障碍（ED）的安全性。
方法
到目前为止，有6名男性符合该方案的入选标准并接受了hMaxi-K基因转移。3人接受了500微克剂量，3人接受了1000微克剂量的基因产物，以裸DNA形式海绵体内注射。计划进行5000微克及更高剂量的给药。
结果
I期试验的主要终点是安全性。基因转移后未发生与药物相关的不良事件或显著的实验室变化。此外，在任何参与者中，精液中基因含量均未达到每微克总DNA 1拷贝的水平。
结论
初步结果表明，在单剂量递增研究中，hMaxi-K离子通道基因转移可安全用于患有ED的男性，且无不良事件发生。
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