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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Detalles Bibliográficos

Principais autores:	Martha Grogan, Farooq H. Sheikh, Brett W. Sperry, Marianne T. Sweetser, Kelley Capocelli, Steven Roblin, Ronald Witteles
Formato:	Artigo
Idioma:	Inglês
Publicado:	Elsevier 2025-09-01
Series:	Molecular Therapy: Nucleic Acids
Assuntos:	MT: Bioinformatics amyloid cardiac amyloidosis hereditary amyloidosis RNA interference small interfering RNAs
Acceso en liña:	http://www.sciencedirect.com/science/article/pii/S2162253125001441
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