Article

Potent and durable gene modulation in heart and muscle with chemically defined siRNAs

2024-10-03

Abstract excerpt

<h4>ABSTRACT</h4> Small interfering RNA (siRNAs) hold immense promise for treating cardiac and muscular diseases, but robust and scalable delivery to these tissues remains a challenge. Recent advances in delivery strategies to muscle include conjugation of biologics (antibody/antibody fragments, peptides), which are currently in clinical development. However, the manufacturing of biologic-siRNA conjugates is a ch...

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Literature Corpus work: e6c7345a-0f88-5f20-8f19-07096d4224b4
DOI: 10.1101/2024.10.01.616183

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