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genetic liver diseases

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RNA-based therapies in liver metabolic diseases

gutjnl · abstract · 2025-08-07 · 1 matching annotations

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hereditary transthyretin amyloidosis genetic liver diseases rare diseases non-human primates small interfering RNA treatments Lipid nanoparticle-mRNA therapeutics RNA-based therapeutics GalNAc conjugation

…r diseases, exemplified by small interfering RNA treatments for hereditary transthyretin amyloidosis, which use liver-targeting strategies such as GalNAc conjugatio…

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genetic liver diseases

Articles mentioning genetic liver diseases

RNA-based therapies in liver metabolic diseases