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Article

Update on the clinical utility of an RNA interference-based treatment: focus on Patisiran

1 Nov 2017

Abstract excerpt

RNA interference (RNAi) is a naturally existing endogenous mechanism for post-transcriptional gene regulation, nowadays commonly utilized for functional characterization of genes and development of potential treatment strategies for diseases. RNAi-based studies for therapy, after being examined for over a decade, are finally in the pipeline for developing a potential treatment for the mutated transthyretin (TTR)...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 29184431
DOI: 10.2147/pgpm.s87945

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