Article

Broad gene expression throughout the mouse and marmoset brain after intravenous delivery of engineered AAV capsids

2020-06-17

Abstract excerpt

<h4>ABSTRACT</h4> Genetic intervention is increasingly explored as a therapeutic option for debilitating disorders of the central nervous system. The safety and efficacy of gene therapies relies upon expressing a transgene in affected cells while minimizing off-target expression. To achieve organ/cell-type specific targeting after intravenous delivery of viral vectors, we employed a Cre-transgenic-based screening...

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Literature Corpus work: ac2c1711-a9c1-50c5-adfc-38458435d923
DOI: 10.1101/2020.06.16.152975

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