Article

<i>In vivo</i> RNA targeting of point mutations via suppressor tRNAs and adenosine deaminases

2017-10-27

Abstract excerpt

<h4>ABSTRACT</h4> Point mutations underlie many genetic diseases. In this regard, while programmable DNA nucleases have been used to repair mutations, their use for gene therapy poses multiple challenges: one, efficiency of homologous recombination is typically low in cells; two, an active nuclease presents a risk of introducing permanent off-target mutations; and three, prevalent programmable nucleases typically...

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Literature Corpus work: 85a6c16c-67e6-54ff-b0f1-cc68b33c8f80
DOI: 10.1101/210278

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