Article

<i>In vivo</i> Treatment of a Severe Vascular Disease via a Bespoke CRISPR-Cas9 Base Editor

2024-11-11

Abstract excerpt

Genetic vascular disorders are prevalent diseases that have diverse etiologies and few treatment options. Pathogenic missense mutations in the alpha actin isotype 2 gene ( ACTA2 ) primarily affect smooth muscle cell (SMC) function and cause multisystemic smooth muscle dysfunction syndrome (MSMDS), a genetic vasculopathy that is associated with stroke, aortic dissection, and death in childhood. Here, we explored g...

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Literature Corpus work: 4bef096b-7e2e-5947-9b54-f6c667e4126f
DOI: 10.1101/2024.11.11.621817

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