Realizing the therapeutic potential of rapid knockdown of transthyretin via RNA interference in transthyretin amyloidosis

RNA interference (RNAi) is a natural biological mechanism that regulates gene expression by degrading a specific target messenger RNA, resulting in reduced production of the target protein. RNAi therapeutics can silence the production of a disease-causing protein in a specific organ or cell type and...

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Autori principali:	Martha Grogan, Farooq H. Sheikh, Brett W. Sperry, Marianne T. Sweetser, Kelley Capocelli, Steven Roblin, Ronald Witteles
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	Elsevier 2025-09-01
Serie:	Molecular Therapy: Nucleic Acids
Soggetti:	MT: Bioinformatics amyloid cardiac amyloidosis hereditary amyloidosis RNA interference small interfering RNAs
Accesso online:	http://www.sciencedirect.com/science/article/pii/S2162253125001441
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