People living with familial cardiomyopathies see gene replacement and gene editing therapies as a promising but uncertain frontier, according to a qualitative study published in BMC Medical Genomics.
The study used a mixed approach: participants first completed a demographic survey and then took part in semi-structured interviews. The researchers developed a codebook both deductively and inductively and applied thematic analysis to the transcripts.
Twenty-one adults completed interviews, all carrying both genetic and clinical diagnoses of familial cardiomyopathy. Seventeen had hypertrophic cardiomyopathy and four had dilated cardiomyopathy. Participants ranged in age from 38 to 80, with a mean age of 57, and 57 percent were female. This was a small, interview-based sample rather than a survey of the broader patient population, so the themes identified describe this group’s perspectives rather than statistically generalizable preferences.
Four main themes shaped how participants reasoned about hypothetically pursuing genetic therapies: perceived clinical severity of their condition, the expected impact on their own disease and potential familial benefit, the safety, mechanism and delivery of the treatment, and a cluster of ethical and social considerations. The severity theme proved especially influential. Those who described a severe clinical presentation expressed greater interest in genetic therapies and a greater willingness to accept risk, particularly when a therapy targeted the root cause of the disease or could halt its progression.
Participants expressed increased interest in genetic therapies particularly when a therapy targeted the root cause of their condition or could halt its progression. Gene therapies designed for cardiomyopathies aim to intervene at the genetic level, and participants who saw their disease as progressive and serious viewed that root-cause approach as worth the uncertainty of a novel intervention. At the same time, participants voiced consistent uncertainties about the safety and long-term consequences of these therapies, reflecting the reality that early-phase trials cannot yet establish durable safety profiles.
Age shaped the discussion in a divided way. Many participants believed that clinical severity, not age, should determine who is prioritized for genetic therapies. Others felt that younger individuals benefit more, since receiving genetic therapy earlier could allow them to avoid lifelong treatment. Family history also weighed heavily: negative experiences that relatives had with cardiomyopathy fed directly into participants’ own decision-making, underscoring how inherited disease makes treatment choices a family matter rather than a purely individual one.
When participants compared genetic therapies with the treatments they already knew, genetic therapies emerged as the most preferred option while implantable cardioverter-defibrillators, or ICDs, ranked as the least preferred. Around one-third of participants said they would hypothetically be interested in joining a clinical trial or adopting a therapy early after FDA approval.
Ethical and social concerns ran through the interviews as well. Participants worried about potential misuse of these therapies, questioned whether they would be distributed equitably across populations, and raised the possibility that editing could inadvertently remove genetic traits that carry some benefit. These concerns echo broader debates in somatic gene therapy but gain particular weight in inherited cardiac disease, where the same variant can affect multiple generations of a family.
The authors conclude that patients’ support and their reservations together highlight the need for clear communication about benefits, risks, uncertainties, and how genetic therapies compare with existing treatment options. They also argue that patients should be included in discussions about the ethical and social impacts of these technologies. As gene replacement and gene editing trials for cardiomyopathies advance, this study offers an early map of what the people these therapies are meant to help actually want to know, fear, and weigh before saying yes.
Subject of Research: Patient perspectives on gene therapies and gene editing for familial cardiomyopathies
Article Title: Patient perspectives on gene therapies and gene editing for familial cardiomyopathies
Article References: Narain, A., Ruiz, K., Wheeler, M., Murtha, R., & Ormond, K. E. (2026). Patient perspectives on gene therapies and gene editing for familial cardiomyopathies. BMC Medical Genomics. https://doi.org/10.1186/s12920-026-02482-8
Image Credits: AI Generated
DOI: 10.1186/s12920-026-02482-8
Keywords: gene therapy, gene editing, familial cardiomyopathy, hypertrophic cardiomyopathy, dilated cardiomyopathy, patient perspectives, qualitative research, thematic analysis, clinical trials, medical genetics, ICD, BMC Medical Genomics
News Source: Juliet Wilcox. (October 28, 2026). Patients weigh promise and peril of gene therapies for inherited heart disease. Scienmag.
