AAV9-ETAM

1. AAV9-ETAM extends lifespan in mice.

   “early ventricular remodeling was prevented and cardiac and skeletal muscle functions were improved, leading to an increased life span of the DmdE4* mice.”

   AnimalsDmdE4* micesingle-doseup to 1 year

   Therapeutic Exon Skipping Through a CRISPR-Guided Cytidine Deaminase Rescues Dystrophic Cardiomyopathy in Vivo

   Circulation·Oct 26, 2021

1. AAV9-ETAM prevents early ventricular remodeling in mice.

   “early ventricular remodeling was prevented and cardiac and skeletal muscle functions were improved, leading to an increased life span of the DmdE4* mice.”

   AnimalsDmdE4* miceheartsingle-doseup to 1 year

   Therapeutic Exon Skipping Through a CRISPR-Guided Cytidine Deaminase Rescues Dystrophic Cardiomyopathy in Vivo

   Circulation·Oct 26, 2021

1. AAV9-ETAM increases DMD in mice (up to 90%).

   “A single-dose administration of AAV9-eTAM instituted >50% targeted exon skipping in the Dmd transcripts and restored up to 90% dystrophin in the heart.”

   AnimalsDmdE4* miceheartsingle-doseup to 1 year

   Therapeutic Exon Skipping Through a CRISPR-Guided Cytidine Deaminase Rescues Dystrophic Cardiomyopathy in Vivo

   Circulation·Oct 26, 2021

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