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Researchers outline three therapeutic approaches targeting troponin T2 for familial dilated cardiomyopathy
Summary
UConn and Jackson Labs researchers presented preclinical work on three approaches—AAV gene replacement, high-activity protein replacement and adenine base editing—aimed at correcting troponin T2-related dilated cardiomyopathy in laboratory models.
A presenter from UConn Health and The Jackson Laboratory described preclinical progress toward therapies for dilated cardiomyopathy caused by pathogenic variants in troponin T2 (TNNT2), including AAV-mediated gene replacement, engineered higher-activity proteins, and base-editing approaches.
The nut graf: Presenters said each approach has a distinct target population and risk–benefit profile: AAV…
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