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Adeno-Associated Virus (AAV) gene therapy for cystic fibrosis: current barriers and recent developments

机译：腺相关病毒（AAV）基因治疗囊性纤维化：当前障碍和最近的发展

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Introduction: Since the cystic fibrosis (CF) gene was discovered in 1989, researchers have worked to develop a gene therapy. One of the most promising and enduring vectors is the AAV, which has been shown to be safe. In particular, several clinical trials have been conducted with AAV serotype 2. All of them detected viral genomes, but identification of mRNA transduction was not consistent; clinical outcomes in Phase II studies were also inconsistent. The lack of a positive outcome has been attributed to a less-than-efficient viral infection by AAV2, a weak transgene promoter and the host immune response to the vector.Areas covered: Herein, the authors focus on AAV gene therapy for CF, evaluating past experience with this approach and identifying ways forward, based on the progress that has already been made in identifying and overcoming the limitations of AAV gene therapy.Expert opinion: Such progress makes it clear that this is an opportune time to push forward toward the development of a gene therapy for CF. Drugs to treat the basic defect in CF represent a remarkable advance but cannot treat a significant cohort of patients with rare mutations. Thus, there is a critical need to develop a gene therapy for those individuals.
机译：介绍：自1989年发现囊性纤维化（CF）基因以来，研究人员已努力开发基因治疗。最有前途和最持久的载体之一是AAV，已被证明是安全的。特别是，已经使用AAV血清型进行了几种临床试验2.所有临床试验2.所有这些临床试验检测到病毒基因组，但鉴定mRNA转导的鉴定不一致; II期研究中的临床结果也不一致。缺乏阳性结果已归因于AAV2，弱转基因启动子和载体对载体的宿主免疫应答的缺乏高效的病毒感染通过这种方法的过去的经验并根据在识别和克服AAV基因治疗的局限性方面取得的进展来确定前进的方式.Expert意见：此类进展明确表示这是推动发展的适当时机CF的基因疗法治疗CF中的基本缺陷的药物代表着一种显着的进步，但不能治疗罕见突变的重要患者群体。因此，需要为这些人发育基因治疗的危急需要。

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