In­tel­li­a's CRISPR ther­a­py like­ly caus­es 'per­ma­nen­t' gene knock­down in AT­TR amy­loi­do­sis

In­tel­lia’s CRISPR-based ther­a­py main­tained re­duc­tions in the lev­els of a mis­fold­ed pro­tein in the nerves of pa­tients with a rare dis­ease called transthyretin amy­loi­do­sis. Func­tion­al …

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