Highlights
Cardiomyopathy gene therapy is shifting from preclinical promise to early human translation, particularly through adeno-associated virus (AAV)-mediated gene replacement and emerging genome-editing platforms.
The most important translational bottleneck is not target discovery alone, but efficient and safe delivery to cardiomyocytes while minimizing liver sequestration, systemic toxicity, and immune barriers.
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For clinical reference; use professional judgment.
