Characterization of RNA Editing and Gene Therapy with a Compact CRISPR-Cas13 in the Retina

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Investigators reported significant transcriptomic signatures of Cas13bt3 expression in retinal cells and show all-in-one adeno-associated virus gene therapy with Cas13bt3 can effectively silence VEGFA mRNA in human retinal organoids and humanized VEGF transgenic mouse models.
[Proceedings of The National Academy of Sciences of The United States of America]
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