缺氧诱导因子-1alpha基因治疗对间歇性脚症患者的步行表现的影响
Mark A Creager1, Jeffrey W Olin, Jill J F Belch
1Division of Cardiovascular Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA. mcreager@partners.org
Circulation
|September 28, 2011
概括
使用Ad2/HIF-1α/VP16的基因治疗没有改善周围动脉疾病患者的行走时间. 这项研究发现,间歇性巴治疗与安慰剂相比没有显著的益处.
科学领域:
- 心血管研究研究心血管研究
- 基因治疗 基因治疗
- 分子医学是分子医学.
背景情况:
- 低氧诱导因子-1α (HIF-1α) 调节细胞对低氧的反应.
- 在临床前模型中,HIF-1α促进了附带血管生长和血液流动.
- 带有间歇性关的外周动脉疾病 (PAD) 在运动时引起腿部疼痛.
研究的目的:
- 评估Ad2/HIF-1α/VP16基因疗法的有效性,以改善PAD患者的行走能力.
- 评估构成性活性HIF-1α对音症状和相关生物标志物的影响.
主要方法:
- 一个双盲随机试验,涉及289名患有听障碍的患者.
- 患者接受了肌肉内Ad2 / HIF-1α / VP16或安慰剂在三个剂量之一.
- 评估包括分级跑步机测试,脚-手臂指数,以及基线和12个月的生活质量评估.
主要成果:
- 在6个月后,在安慰剂和任何Ad2/HIF-1α/VP16剂量组之间没有观察到峰值行走时间的显著差异.
- 音发病时间,脚-手臂指数和生活质量指标也没有显著改善.
- 该研究发现,这种基因治疗对安慰剂没有统计学上显著的益处.
结论:
- 肌肉内Ad2/HIF-1α/VP16基因疗法不是间歇性听症的有效治疗方法.
- 可能需要进一步的研究来探索其他治疗策略.
- 临床试验NCT00117650没有证明这种基因治疗方法的有效性.
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