AAV9-mediated <i>KCNH2</i> suppression-replacement gene therapy in a transgenic rabbit model of type 1 short QT syndrome
This proof-of-concept study is the first to demonstrate the efficacy of gene therapy for SQT1 in a medium-sized animal model. KCNH2-SupRep gene therapy successfully corrected the pathologic phenotype in vivo, ex vivo, and at cellular levels in transgenic SQT1 rabbits.
Authors
- Nicolas Christoforou (ORCID: https://orcid.org/0000-0003-4594-6919)
- András Horváth (ORCID: https://orcid.org/0000-0002-8974-0771)
- Julien Louradour (ORCID: https://orcid.org/0000-0002-0649-4975)
- C.S. John Kim
- Manfred Zehender
- Dan Ye (ORCID: https://orcid.org/0000-0001-5419-0522)
- Michael Ackerman (ORCID: https://orcid.org/0000-0002-0595-8942)
- Manuel Egle (ORCID: https://orcid.org/0009-0000-6368-7397)
- Jordi Heijman (ORCID: https://orcid.org/0000-0002-1418-108X)
- Fabien Praz (ORCID: https://orcid.org/0000-0001-5416-165X)
- Nicolò Alerni (ORCID: https://orcid.org/0000-0002-4929-3479)
- Miriam Barbieri (ORCID: https://orcid.org/0009-0002-4589-5998)
- Sahej Bains (ORCID: https://orcid.org/0000-0002-1357-7656)
- Gabriel Brooks (ORCID: https://orcid.org/0000-0003-0207-5527)
- David J. Tester (ORCID: https://orcid.org/0000-0003-4554-2244)
- Lisa R. Keyes
- Andreas Haeberlin (ORCID: https://orcid.org/0000-0002-9283-0110)
- Jane Owens (ORCID: https://orcid.org/0000-0002-4916-7202)
- Saranda Nimani (ORCID: https://orcid.org/0000-0002-9298-3678)
- Michael A. Barry (ORCID: https://orcid.org/0000-0002-8568-5664)
- Katja E. Odening (ORCID: https://orcid.org/0000-0001-6999-841X)
- Jason Pinkstaff
- Jan Christoph (ORCID: https://orcid.org/0000-0003-4369-3591)
- Rubén López (ORCID: https://orcid.org/0000-0002-3802-3158)
- Balázs Ördög (ORCID: https://orcid.org/0000-0002-8971-3079)
- Chiara Parodi (ORCID: https://orcid.org/0000-0002-2689-2049)
- Mariana G. Bego (ORCID: https://orcid.org/0000-0002-1963-5503)
- Jacqulyn Jurgensen
- Lucilla Giammarino (ORCID: https://orcid.org/0000-0002-2100-0160)
- Daniela Casoni (ORCID: https://orcid.org/0000-0001-8852-2134)
- Olgica Beslac
- Wei Zhou (ORCID: https://orcid.org/0000-0001-7719-0859)
- Michael Brunner
- Stefan Meier
- Stefanie Perez‐Feliz (ORCID: https://orcid.org/0009-0009-2880-2367)
- Lluis Matas
- L.G. Garcia Casalta
- Varjany Vashanthakumar
Institutions
- University of Bern (CH)
- Heart Rhythm Society (US)
- Bern University of Applied Sciences (CH)
- Mayo Clinic (US)
- University of Freiburg (DE)
- University of California, San Francisco (US)
- Pfizer (United States) (US)
- Medical University of Graz (AT)
- Maastricht University Medical Centre (NL)
- University Hospital of Bern (CH)
- University Medical Center Freiburg (DE)
- Maastricht University (NL)
- Alexion Pharmaceuticals (United States) (US)
- Vector (United States) (US)
- Mayo Clinic in Florida (US)
- St. Josefskrankenhaus Freiburg (DE)
- Solidus Biosciences (United States) (US)
- Alexion Pharma (Switzerland) (CH)
Publication Details
- Journal
- European Heart Journal
- Published
- 2025-08-15
- DOI
- https://doi.org/10.1093/eurheartj/ehaf660
- Citations
- 2
- Primary Topic
- Cardiac electrophysiology and arrhythmias
- Type
- article
- Field-Weighted Citation Impact
- 2.78
Funders
- University of Bern