Somatic gene editing for inherited cardiomyopathy was viewed with optimism and trust by 18 stakeholders, who emphasized its potential as a root cause fix and highlighted societal benefits.
There is a rare degree of alignment and optimism among patients, professionals, and regulators regarding the responsible development of somatic gene editing for inherited cardiomyopathies.
This qualitative study aims to understand the moral attitudes of key stakeholders toward the development of somatic gene editing for inherited cardiomyopathy. Patients are waiting on effective treatment for inherited cardiomyopathies, including those caused by PLN and LMNA mutations. Somatic gene editing, particularly prime and base editing, is being explored as a potential therapeutic strategy. Development of these technologies requires insight into the perspectives of stakeholders directly affected by or involved in their translation. Semi-structured interviews were conducted with eighteen stakeholders in The Netherlands, including PLN and LMNA-mutation carriers, clinicians, laboratory researchers, and regulatory officers. Interview transcripts were thematically analysed to foster collaborative development of higher-order themes. Three central themes were identified. First, participants emphasized the importance of "the right treatment for the right person at the right time," reflecting a mixture of cautious pragmatism and hopeful anticipation of meeting patients' unmet medical need. Second, respondents highlighted potential societal benefits, like cost savings and technological spill-over, as justifications for continued development of somatic gene editing for inherited cardiomyopathy. Third, stakeholders expressed notable trust, not only in the promise of somatic gene editing itself but also in the broader process of scientific and clinical advancement. Respondents recognized somatic gene editing as a potential root cause fix, but attitudes spanned from considering it the superior option to maintaining a more ambivalent stance. Importantly, expressed concerns were framed as challenges to overcome rather than grounds for rejection. This convergence of optimism and trust across stakeholder groups underscores a rare degree of alignment between patients, professionals, and regulators; an alignment that demands careful stewardship if enthusiasm is to translate into responsible development and implementation of somatic gene editing for inherited cardiomyopathy.
Simons et al. (Mon,) conducted a other in Inherited cardiomyopathy (n=18). Somatic gene editing (attitudes toward) was evaluated on Moral attitudes and perspectives on somatic gene editing. Somatic gene editing for inherited cardiomyopathy was viewed with optimism and trust by 18 stakeholders, who emphasized its potential as a root cause fix and highlighted societal benefits.