Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells.
Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells.
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作者：
基因转移有可能作为一种一次性的治疗方法，减少病毒载量，保护免疫系统，并避免终身高活性抗逆转录病毒治疗。这项研究是第一项随机、双盲、安慰剂对照、II期细胞递送基因转移临床试验，在74名接受达特/vpr特异性抗HIV核酶（OZ 1）或安慰剂递送自体CD 34+造血祖细胞的HIV-1感染成人中进行。没有OZ 1相关的不良事件。在主要终点（第47周和第48周的平均值），OZ 1组和安慰剂组之间的病毒载量没有统计学差异，但OZ 1组第40-48周和第40-100周的时间加权曲线下面积显著较低。在整个100周内，OZ 1组的CD 4+淋巴细胞计数较高。这项研究首次表明，细胞递送的基因转移在HIV患者中是安全和生物活性的，可以作为常规治疗产品开发。
Gene transfer has potential as a once-only treatment that reduces viral load, preserves the immune system, and avoids lifetime highly active antiretroviral therapy. This study, the first randomized, double-blind, placebo-controlled, phase II cell-delivered gene transfer clinical trial, was conducted in 74 HIV-1 infected adults who received a tat/vpr specific anti-HIV ribozyme (OZ1) or placebo delivered in autologous CD34+ hematopoietic progenitor cells. There were no OZ1-related adverse events. There was no statistical difference in viral load between the OZ1 and placebo group at the primary end-point (average at weeks 47 and 48) but time weighted areas under the curve from weeks 40-48 and 40-100 were significantly lower in the OZ1 group. Throughout the 100 weeks, CD4+ lymphocyte counts were higher in the OZ1 group. This study provides the first indication that cell-delivered gene transfer is safe and biologically active in HIV patients and can be developed as a conventional therapeutic product.
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影响因子：
20.3
作者：
Kohn, DB;Bauer, G;Church, J
通讯作者：
Church, J
DOI：
10.2741/macpherson
发表时间：
1999-06-01
期刊：
Frontiers in Bioscience
影响因子：
--
作者：
MacPherson, Janet L.;Ely, Julie A.;Symonds, Geoff P.
通讯作者：
Symonds, Geoff P.
DOI：
10.1073/pnas.94.22.12133
发表时间：
1997-10-28
影响因子：
11.1
作者：
Malech, HL;Maples, PB;Gallin, JI
通讯作者：
Gallin, JI
影响因子：
4.2
作者：
Mautino, MR;Morgan, RA
通讯作者：
Morgan, RA
DOI：
10.1073/pnas.0705474104
发表时间：
2007-08-07
影响因子：
11.1
作者：
An, Dong Sung;Donahue, Robert E.;Chen, Irvin S. Y.
通讯作者：
Chen, Irvin S. Y.