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Research Review

Showing 1–1 of 1 articles · Keyword: Cardiomyopathy Clear keyword

Current cardiology reports2026ReviewNon-viral Gene Delivery

1. Viral Gene Therapy for Danon Disease: From LAMP2 Biology to Clinical Translation

Gunes Ea, Patel U, Iyer S, Taub A, Lee-Guest E, Cline S, Et Al.

Danon disease is a rare, highly penetrant and morbid X-linked cardioskeletal myopathy caused by loss-of-function variants in the lysosome-associated membrane protein 2 (LAMP2) gene. We review the rationale for LAMP2B gene replacement, the basis for adeno-associated virus serotype 9 (AAV9) vector selection, the…

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