Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2022 Jul;30(7-8):343-344.
doi: 10.1007/s12471-022-01665-z. Epub 2022 Mar 2.

Will our cardiomyopathy patients accept gene therapy?

P A Doevendans  1   2 , C Kupatt  3 , M Giacca  4 , P Glijnis  5
Affiliations
Review

Will our cardiomyopathy patients accept gene therapy?

P A Doevendans et al. Neth Heart J. 2022 Jul.

Abstract

Novel techniques such as gene therapy are becoming available in an attempt to cure inherited diseases. Before these new therapies can be offered to patients, we need to be aware of potential reservations or objections, not only from patients and their surroundings but also from the public. In addition, legal issues and costs need attention before curative gene therapy can be applied in the clinic. As this therapeutic approach is closer to becoming a reality, now is the right time to start the debate.

Keywords: Cardiomyopathy; Gene editing; Viral vectors.

PubMed Disclaimer

Conflict of interest statement

P.A. Doevendans, C. Kupatt, M. Giacca and P. Glijnis declare that they have no competing interests.

References

    1. Koblan LW, Erdos MR, Wilson C, et al. In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice. Nature. 2021;589:608–614. doi: 10.1038/s41586-020-03086-7. - DOI - PMC - PubMed
    1. Ganguly P. www.genome.gov/news/news-release/DNA-editing-method-shows-promise-to-tre.... Accessed 14 Jan 2022.
    1. Moretti A, Fonteyne L, Giesert F, et al. Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy. Nat Med. 2020;26:207–214. doi: 10.1038/s41591-019-0738-2. - DOI - PMC - PubMed
    1. Bainbridge JWB, Mehat MS, Sundaram V, et al. Long-term effect of gene therapy on Leber’s congenital amaurosis. N Engl J Med. 2015;372:1887–1897. doi: 10.1056/NEJMoa1414221. - DOI - PMC - PubMed
    1. Mack HG, Chen FK, Grigg J, et al. Perspectives of people with inherited retinal diseases on ocular gene therapy in Australia: protocol for a national survey. BMJ Open. 2021;11:e048361. doi: 10.1136/bmjopen-2020-048361. - DOI - PMC - PubMed