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    Home > Active Ingredient News > Immunology News > NEJM: CRISPR-Cas9 in vivo gene editing to treat transthyretin amyloidosis

    NEJM: CRISPR-Cas9 in vivo gene editing to treat transthyretin amyloidosis

    • Last Update: 2021-08-14
    • Source: Internet
    • Author: User
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    Transthyretin amyloidosis, also called ATTR amyloidosis, is a life-threatening disease characterized by the gradual accumulation of misfolded transthyretin (TTR) in tissues, mainly in the nerves and heart


    NTLA-2001 is an in vivo gene editing therapeutic agent designed to treat ATTR amyloidosis by reducing the concentration of TTR in serum






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