Donnelley Martin, Parsons David, Prichard Ivanka
Robinson Research Institute, University of Adelaide, Adelaide, South Australia.
Adelaide Medical School, University of Adelaide, Adelaide, South Australia.
Expert Opin Biol Ther. 2023 Jan;23(1):103-113. doi: 10.1080/14712598.2022.2150544. Epub 2022 Dec 6.
Airway gene therapy could produce lasting benefit for cystic fibrosis (CF) lung disease, however patient and public support is critical for successful adoption.
Two separate quantitative online surveys were conducted to examine perceptions towards airway gene therapy for CF among people with CF, their families, and members of the public. Data was collected from a total of 213 participants across both studies, with 43 having a diagnosis of CF, 122 having a family member with CF, and 135 knowing someone with CF.
Participants in both studies displayed positive perceptions towards gene therapy and were supportive of involvement in CF gene therapy trials. Around 50% hoped gene therapy could provide a cure. In Study 1 gene therapy was the most important research area, but in Study 2 this was new daily drugs. Almost all thought gene therapy was still required even if modulators already improved quality of life.
The factors that influence acceptance, whether trials would be positively viewed, and whether individuals with CF are receptive to gene therapy, are essential to determine prior to clinical trials. Our findings indicate people have positive opinions about airway gene therapy for CF, but further education is vital.
气道基因治疗可能会给囊性纤维化（CF）肺部疾病带来持久益处，然而患者和公众的支持对于其成功应用至关重要。
开展了两项独立的定量在线调查，以研究CF患者、其家人及公众对CF气道基因治疗的看法。两项研究共收集了213名参与者的数据，其中43人被诊断为CF，122人有家庭成员患CF，135人认识CF患者。
两项研究的参与者对基因治疗均持积极看法，并支持参与CF基因治疗试验。约50%的人希望基因治疗能实现治愈。在研究1中，基因治疗是最重要的研究领域，但在研究2中则是新型日常药物。几乎所有人都认为，即便调节剂已改善了生活质量，基因治疗仍不可或缺。
在临床试验之前，确定影响接受度的因素、试验是否会被积极看待以及CF患者是否接受基因治疗至关重要。我们的研究结果表明，人们对CF气道基因治疗持积极态度，但进一步的教育至关重要。
