关于眼部基因疗法潜在参与者观点的多国调查

IF 4.6 3区 医学 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY Gene Therapy Pub Date : 2024-04-02 DOI:10.1038/s41434-024-00450-4
Alexis Ceecee Britten-Jones, Myra B. McGuinness, Fred K. Chen, John R. Grigg, Heather G. Mack, Lauren N. Ayton
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引用次数: 0

摘要

眼部基因疗法发展迅速,了解患者的观点对于确定未来的治疗选择和研究方向至关重要。这项国际横断面调查评估了患有遗传性视网膜疾病(IRD)的潜在接受者对眼部基因疗法的了解、态度和看法。调查工具包括基因治疗态度(AGT-Eye)、EQ-5D-5L、美国国家眼科研究所视觉功能问卷(NEI-VFQ-25)和患者对临床试验的态度(PACT-22)工具。这项研究包括来自 35 个国家的 496 名参与者(89% 患有 IRD 的成年人;11% 的父母/监护人/照看者)的回复,其中大部分来自美国(69%)和英国(11%)。大多数参与者(90%)表示,如果有基因疗法,他们很可能会接受,尽管只有 45% 的人认为他们对基因疗法有充分的了解。信息的主要来源是研究登记(60% 的参与者)和互联网(61%)。与我们最近发布的澳大利亚全国IRD患者调查(n = 694)数据相比,美国受访者对基因治疗结果的了解程度更高,澳大利亚受访者对基因治疗价值的认知度更高。解决有关治疗结果和财务影响的知识差距将是确保知情同意、促进共同决策以及最终临床采用基因疗法的核心。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

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A multinational survey of potential participant perspectives on ocular gene therapy
Amidst rapid advancements in ocular gene therapy, understanding patient perspectives is crucial for shaping future treatment choices and research directions. This international cross-sectional survey evaluated knowledge, attitudes, and perceptions of ocular genetic therapies among potential recipients with inherited retinal diseases (IRDs). Survey instruments included the Attitudes to Gene Therapy-Eye (AGT-Eye), EQ-5D-5L, National Eye Institute Visual Functioning Questionnaire (NEI-VFQ-25), and Patient Attitudes to Clinical Trials (PACT-22) instruments. This study included 496 participant responses (89% adults with IRDs; 11% parents/guardians/carers) from 35 countries, with most from the United States of America (USA; 69%) and the United Kingdom (11%). Most participants (90%) indicated they would likely accept gene therapy if it was available, despite only 45% agreeing that they had good knowledge of gene therapy. The main sources of information were research registries (60% of participants) and the internet (61%). Compared to data from our recently published Australian national survey of people with IRDs (n = 694), USA respondents had higher knowledge of gene therapy outcomes, and Australian respondents indicated a higher perceived value of gene therapy treatments. Addressing knowledge gaps regarding outcomes and financial implications will be central to ensuring informed consent, promoting shared decision-making, and the eventual clinical adoption of genetic therapies.
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来源期刊
Gene Therapy
Gene Therapy 医学-生化与分子生物学
CiteScore
9.70
自引率
2.00%
发文量
67
审稿时长
4-8 weeks
期刊介绍: Gene Therapy covers both the research and clinical applications of novel therapeutic techniques based on a genetic component. Over the last few decades, significant advances in technologies ranging from identifying novel genetic targets that cause disease through to clinical studies, which show therapeutic benefit, have elevated this multidisciplinary field to the forefront of modern medicine.
期刊最新文献
Gene-based therapy for the treatment of spinal muscular atrophy types 1 and 2 : a systematic review and meta-analysis. PCRX-201, a novel IL-1Ra gene therapy treatment approach for low back pain resulting from intervertebral disc degeneration. Bridging gene therapy and next-generation vaccine technologies. Retraction Note: Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations. Non-replicative herpes simplex virus genomic and amplicon vectors for gene therapy - an update.
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