心臓発作後の非ヒト霊長類における遺伝子治療による機能回復(Gene Therapy Restores Functionality in Non-Human Primates After Heart Attacks)

2025-08-28 デューク大学

<関連情報>

• https://pratt.duke.edu/news/heart-gene-therapy-nhp/
• https://www.ahajournals.org/doi/10.1161/CIRCRESAHA.125.326570

BacNav遺伝子治療が人工心筋梗塞ヒト心筋および非ヒト霊長類の機能を改善
BacNa_v Gene Therapy Improves Function of Infarcted Engineered Human Myocardium and NHPs

Tianyu Wu, Nicole G.Z. Tee, Yiu Yan Siu, Anna Tornatore, Abhishek Bhattacharjee, James Koconis, Sze Jie Loo, Liping Su, Binjie Li, Lei Ye, and Nenad Bursac
Circulation Research  Published: 1 August 2025
DOI:https://doi.org/10.1161/CIRCRESAHA.125.326570

Current clinical therapies for myocardial infarction (MI) and sudden cardiac death show limited efficacy.¹ The ability to enhance amplitudes of peak sodium (Na⁺) current and calcium (Ca²⁺) transient in cardiomyocytes could uniquely prevent arrhythmias and improve the contractile function of infarcted hearts.

Previously, we leveraged the small size of engineered prokaryotic voltage-gated Na⁺ channels (BacNa_v, <1 kb) to overcome the adeno-associated virus (AAV) packaging constraint and demonstrated that BacNa_v expression can directly enhance cardiac excitability.² Here, we investigated whether cardiomyocyte-specific BacNa_v expression can provide both antiarrhythmic and inotropic benefits to the injured heart.