研究人员追求单剂量基因疗法治疗可卡因成瘾

梅奥诊所(Mayo Clinic)的研究人员正在寻求批准一种治疗可卡因成瘾的全新方法，首次进行单剂量基因疗法的人体试验。为了证明这种方法的安全性和有效性，他们已经成功地将一种酶的编码基因导入小鼠体内，这种酶可以将可卡因代谢成无害的副产品。该研究发表于人类基因治疗。

斯蒂芬Brimijoin和来自Mayo Clinic，Rochester，Mn的同事，共同定制了题为“用于人类可卡因水解基因治疗的重组AAV8载体的全身安全性：小鼠的良好实验室实践临床前研究。”In advance of filing for an Investigational New Drug Application with the U.S. Food and Drug Administration, which would allow for human testing, the researchers needed to show the systemic safety of their recombinant adeno-associated viral (AAV) 8 vector, which targets its therapeutic gene payload to the liver. They showed a total lack of viral vector-related adverse effects in both cocaine-experienced and cocaine-naïve mice at different doses. In fact, mice who received the gene therapy followed by daily cocaine injections had much less tissue pathology than those mice who received daily cocaine injections but did not have the gene therapy.

“物质使用障碍在美国和其他工业化国家提供了一个巨大的公共卫生问题，”主编R. Flotte，MD，Celia和Isaac Haidak医学教育和院长教授，Progost和行政副总裁教授。马萨诸塞大学医学院，伍斯特，马。“把创新的基因治疗的力量施加在这个问题上呈现出一种令人兴奋的新方法。”

---

---