Will our cardiomyopathy patients accept gene therapy?

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Will our cardiomyopathy patients accept gene therapy?
 
Doevendans, P. A.; Kupatt, C.; Giacca, M.; Glijnis, P.
(2022) Netherlands Heart Journal, volume 30, issue 7-8, pp. 343 - 344
(Article)
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 ... read more
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Keywords: Cardiomyopathy, Gene editing, Viral vectors, Cardiology and Cardiovascular Medicine
DOI: https://doi.org/10.1007/s12471-022-01665-z
ISSN: 1568-5888
Publisher: Bohn Stafleu van Loghum
Note: Funding Information: The first ocular gene therapy that was approved by the American Food and Drug Administration, voretigene neparvovec-rzyl (Luxturna; Spark Therapeutics, USA), uses an AAV2 vector to deliver a functional copy of the RPE65 gene for the treatment of Leber congenital amaurosis type 2. This autosomal recessive inherited eye disease leads to impaired vision or even blindness at birth, but the phenotype is variable. Clinical trials to evaluate safety and effectiveness are ongoing, [] supported by the Foundation Fighting Blindness ( www.fightingblindness.org ). While this therapy does not provide a cure, it leads to prolonged normal protein expression. In Australia, a protocol has been approved to evaluate the ethics of ocular gene therapy []. The research group involved is in the process of developing a new ‘Attitudes to Gene Therapy for the Eye’ tool. Funding Information: P. A. Doevendans has received a grant from the Netherlands Organisation for Health Research and Development (ZonMw) (PSIDER-Heart) and the Leducq Foundation (CUREPLaN). Publisher Copyright: © 2022, The Author(s).
(Peer reviewed)