争论还在持续对人类免疫系统的作用,阻断治疗基因传递使用AAV载体

假设记忆T细胞形成针对儿童感染可能造成的损失的表达治疗基因通过病毒载体被热议,但最近的临床试验的腺相关病毒(AAV)基于矢量的基因疗法专门为了避免T细胞反应AAV显示正确的使人衰弱的疾病,如血友病的能力在某些病人。科学家们提出这个假设讨论正在进行的争论和发展方法克服AAV-vector-induced T细胞反应的一篇文章中人类基因治疗。

Hildegund Ertl、威斯塔研究所和凯瑟琳高,引发疗法,费城,宾夕法尼亚州,是文章的合著者题为“AAV Capsid-Specific t细胞反应的影响对设计和结果的临床与重组腺相关病毒载体基因转移试验:一个不断发展的争议。”They describe the evidence that first led them to look more closely at how T cell responses could interfere in successful therapeutic gene delivery using AAV vectors and why their hypothesis was initially met with much skepticism. The researchers also discuss some of the strategies developed that have shown that it is possible to avoid a T-cell response. These include immunosuppression, use of rare AAV serotypes that the immune system would not typically have previous exposure to, or modified versions of AAV vectors that can deliver and achieve expression of sufficient levels of the therapeutic gene without eliciting an免疫反应。

“理解免疫反应AAV载体将充分和安全开采的关键疾病如血友病的治疗潜力,”特伦斯·r·Flotte主编说,医学博士,西莉亚和艾萨克Haidak医学教育教授和院长,院长,和常务副总理,马萨诸塞大学医学院,伍斯特马。