Genethon, BP60, 91002 Evry Cedex, France.
Macromol Biosci. 2011 May 12;11(5):590-4. doi: 10.1002/mabi.201000463. Epub 2011 Feb 17.
Muscle is an important and attractive target for gene therapy. Recent findings have shown that neutral amphiphilic triblock copolymers with a PEO-PPO-PEO arrangement significantly increase muscle transfection as compared to naked DNA. We were interested in evaluating whether reverse Pluronics (PPO-PEO-PPO) also possess transfection properties. Therefore, we measured the in vitro and in vivo transfection activity of 25R2 and 25R4, two copolymers that differ by their hydrophilic/hydrophobic balance. The results show that 25R2 significantly increases the transfection level in muscle compared to naked DNA. Taken together, this work demonstrates that the reverse Pluronic 25R2 possesses interesting properties for in vivo transfection.
肌肉是基因治疗的一个重要且有吸引力的靶标。最近的研究结果表明,与裸露 DNA 相比,具有 PEO-PPO-PEO 排列的中性两亲性嵌段共聚物可显著提高肌肉转染效率。我们有兴趣评估反向普朗尼克(PPO-PEO-PPO)是否也具有转染特性。因此,我们测量了两种共聚物 25R2 和 25R4 的体外和体内转染活性,这两种共聚物的亲水/疏水平衡不同。结果表明,与裸露 DNA 相比,25R2 可显著提高肌肉中的转染水平。总之,这项工作表明,反向普朗尼克 25R2 具有用于体内转染的有趣特性。