Key result
Weekly IV AC modRNA preserves cardiac function and prevents fibrosis in chronic Dox-induced cardiotoxicity models.
Why the study?
Doxorubicin use is clinically limited by dose-accumulative cardiotoxicity, and therapeutic modified mRNA applications to protect the heart have been hindered by the need for invasive delivery.
Does intravenous delivery of acid ceramidase modified mRNA using a cardiomyocyte-selective platform prevent doxorubicin-induced cardiotoxicity in preclinical models?
Population
Dox-treated human iPSC-derived CMs and chronic Dox-induced cardiotoxicity models
Comparison
IV AC modRNA via cmSMRTs 143-122 vs control
Design
Preclinical in vitro and in vivo study
Authors
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Supports testing of cardiomyocyte-targeted mRNA in doxorubicin recipients; leaves open clinical translation from animal models.
Does intravenous delivery of acid ceramidase modified mRNA using a cardiomyocyte-selective platform prevent doxorubicin-induced cardiotoxicity in preclinical models?
A novel cardiac-selective mRNA delivery platform for acid ceramidase prevents doxorubicin-induced cardiotoxicity in preclinical models without reducing chemotherapeutic efficacy.
Yoo et al. (2026) studied this question. Weekly IV delivery of AC modified mRNA preserved cardiac function and prevented dysfunction, fibrosis, and atrophy in chronic Dox-induced cardiotoxicity models.
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