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ACTIVITY OF FOLLOW-ON DOSING FOR AN INVESTIGATIONAL IN VIVO CRISPR-BASED LNP THERAPY IN TRANSTHYRETIN AMYLOIDOSIS

Jorg Taubel,Ed Gane,Marianna Fontana,Justin Kao,David Adams,Bjorn Pilebro,Michael Maitland, Derek Smith, Michael D. Pickard,Yuanxin Xu,Adam Amaral,Carri Boiselle, Rebecca Lescarbeau,David Gutstein,Liron Walsh,Julian D. Gillmore

JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM（2024）

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Key words
TTR,amyloidosis,CRISPR,cardiomyopathy,gene therapy
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