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Article

Innovations in RNA therapy for hemophilia

21 Jul 2023

Abstract excerpt

Given the shortcomings of current factor-, nonfactor-, and adeno-associated virus gene-based therapies, the recent advent of RNA-based therapeutics for hemophilia is changing the fundamental approach to hemophilia management. From small interfering RNA therapeutics that knockdown clot regulators antithrombin, protein S, and heparin cofactor II, to CRISPR/Cas9 gene editing that may personalize treatment, improved...

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PMID: 37478403
DOI: 10.1182/blood.2022018661

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