PURPOSE Genome editing holds both tremendous therapeutic promise and significant potential risk. Sickle cell disease (SCD), the most commonly inherited blood disorder, is a frontline candidate for the clinical applications of this tool. However, there is limited knowledge of patient community values and concerns regarding this new technology. This study aims to investigate the perspectives of three key decision-makers (patients, parents, and physicians) toward participation in future CRISPR-mediated somatic genome editing clinical trials. METHODS We utilized a mixed-methods approach, involving an educational video tool, two-part survey, and 15 moderated, audio-recorded focus groups, which were conducted in seven U.S. cities. RESULTS Study participants expressed hope that genome editing technology would rechart the course for SCD, but concerns related to involvement burden, uncertainty of clinical outcomes, and equity in access were identified. Major themes emerged from the focus groups: facilitators of, and barriers to, participation in future somatic genome editing clinical trials; information pertinent to the decision-making process; persons from whom participants would seek counsel before making a decision; and recommendations for the research community on meaningful engagement as clinical trials are designed and approved. CONCLUSION The advent of genome editing has renewed hope for the SCD community, but caution tempers this optimism.

中文翻译：

---

CRISPR 关注镰状细胞病社区内利益相关者对体细胞基因组编辑的态度和信念。

目的 基因组编辑具有巨大的治疗前景和巨大的潜在风险。镰状细胞病 (SCD) 是最常见的遗传性血液疾病，是该工具临床应用的一线候选者。然而，对患者社区价值观和对这项新技术的担忧的了解有限。本研究旨在调查三位关键决策者（患者、父母和医生）对参与未来 CRISPR 介导的体细胞基因组编辑临床试验的看法。方法 我们采用了一种混合方法，包括一个教育视频工具、两部分调查和 15 个有主持的、录音的焦点小组，这些小组在美国的七个城市进行。结果 研究参与者表示希望基因组编辑技术能够重新定义 SCD 的进程，但确定了与参与负担、临床结果的不确定性和获取公平性有关的担忧。焦点小组提出的主要主题：参与未来体细胞基因组编辑临床试验的促进者和障碍；与决策过程相关的信息；参与者在做出决定之前会寻求咨询的人；以及在设计和批准临床试验时为研究界提供有意义的参与的建议。结论 基因组编辑的出现为 SCD 社区重新燃起了希望，但谨慎缓和了这种乐观情绪。参与未来的体细胞基因组编辑临床试验；与决策过程相关的信息；参与者在做出决定之前会寻求咨询的人；以及在设计和批准临床试验时为研究界提供有意义的参与的建议。结论 基因组编辑的出现为 SCD 社区重新燃起了希望，但谨慎缓和了这种乐观情绪。参与未来的体细胞基因组编辑临床试验；与决策过程相关的信息；参与者在做出决定之前会寻求咨询的人；以及在设计和批准临床试验时为研究界提供有意义的参与的建议。结论 基因组编辑的出现为 SCD 社区重新燃起了希望，但谨慎缓和了这种乐观情绪。