Cardiac gene therapy makes a comeback

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Cardiac gene therapy makes a comeback
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Gene therapy

Nature Medicine (2025)Cite this article

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The first-in-human trial of AAV2i8-I1c (AB-1002) — an adenovirus vector that targets a protein phosphatase inhibitor to cardiac muscle — suggests a cautious return of cardiac gene therapy after earlier setbacks, with a new target, dose optimization and improved vector design shaping future directions.

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Fig. 1: Two key strategies for targeting calcium handling in clinical trials of gene therapy in heart failure.

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Authors and Affiliations

  1. Department of Internal Medicine V, University Hospital Schleswig-Holstein, University of Kiel, Kiel, Germany

    Oliver J. Müller, Susanne Hille & Anca Kliesow Remes

  2. German Centre for Cardiovascular Research (DZHK), partner site Hamburg/Kiel/Lübeck, Kiel, Germany

    Oliver J. Müller, Susanne Hille & Anca Kliesow Remes

Authors

  1. Oliver J. Müller
  2. Susanne Hille
  3. Anca Kliesow Remes

Corresponding author

Correspondence to Oliver J. Müller.

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Competing interests

The authors declare no competing interests.

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Cite this article

Müller, O.J., Hille, S. & Kliesow Remes, A. Cardiac gene therapy makes a comeback. Nat Med (2025). https://doi.org/10.1038/s41591-025-04002-0

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