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June 26, 2017European Heart Journal134 citations

Identification of a targeted and testable antiarrhythmic therapy for long-QT syndrome type 2 using a patient-specific cellular model

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AMAshish MehtaCRChrishan J. A. RamachandraPSPritpal Singh

Structured PICO

Does lumacaftor rescue the pathological phenotype in LQT2 iPSC-CMs?

P
Population
Patient-specific cellular model (induced pluripotent stem cell-derived cardiomyocytes, iPSC-CMs) from patients with long-QT syndrome type 2 (LQT2), particularly Class 2 mutations
I
Intervention
Lumacaftor
O
Outcome
Rescue of the pathological phenotype of LQT2 iPSC-CMssurrogate

Lumacaftor may represent a novel therapeutic option for LQT2 patients not protected by beta-blockers by rescuing the cellular pathological phenotype.

Abstract

Lumacaftor, a drug already in clinical use, can rescue the pathological phenotype of LQT2 iPSC-CMs, particularly those derived from Class 2 mutated patients. Our results suggest that the use of LUM in LQT2 patients not protected by β-blockers is feasible and may represent a novel therapeutic option.

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Cite This Study

Mehta et al. (2017) studied this question.

synapsesocial.com/papers/69d9d9d68988aeabbe686319https://doi.org/10.1093/eurheartj/ehx394
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