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Article

AAV-TNNI3 rescues an experimental murine Tnni3 mutation resulting in thin filament mediated DCM.

Journal of molecular and cellular cardiology - 1 Mar 2026

Bushway Paul J, Feng Wei, Cruz Marina Sampaio De Menezes, Maisel Sofie, Shathaya Aysen, Rao Pranita, Patel Umang, Park Jinsun, Chen Chao, Tang Zhiyuan, Gunes Betul, Gunes Eren, Ye Mao, Gu Yusu, Adler Eric

Abstract excerpt

Cardiac thin filament mutations in TNNI3 are associated with up to 3 % of hypertrophic (HCM) cardiomyopathy cases and contribute to severe restrictive (RCM) and dilated (DCM) cardiomyopathy caseloads. As such, thin filament cardiomyopathy mediated by TNNI3 mutations is an orphan disease with unmet therapeutic need. Gene therapy is one approach to addressing orphan disease but has been restricted to the repletion...

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Identifiers

PMID: 41494578
DOI: 10.1016/j.yjmcc.2026.01.001

Genes

TNNI3

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 8 Sept 2026.

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