AAV9-mediated MYBPC3 gene therapy with optimized expression cassette enhances cardiac function and survival in MYBPC3 cardiomyopathy models.
Greer-Short A, Greenwood A, Leon EC, Qureshi TN, von Kraut K, Wong J, Tsui JH, Reid CA, Cheng Z, Easter E, Yang J, Ho J, Steltzer S, Budan A, Cho M, Chandrakumar R, Cisne-Thompson O, Feathers C, Chung TW, Rodriguez N, Jones S, Alleyne-Levy C, Liu J, Jing F, Prince WS, Lin J, Ivey KN, Tingley WG, Hoey T, Lombardi LM.
Greer-Short A, et al. Among authors: easter e.
Nat Commun. 2025 Mar 4;16(1):2196. doi: 10.1038/s41467-025-57481-7.
Nat Commun. 2025.
PMID: 40038304
Free PMC article.