Article

Improved specificity and efficiency of <i>in vivo</i> adenine base editing therapies with hybrid guide RNAs

2024-04-22

Abstract excerpt

Phenylketonuria (PKU), pseudoxanthoma elasticum (PXE), and hereditary tyrosinemia type 1 (HT1) are autosomal recessive disorders linked to the PAH , ABCC6 , and FAH and HPD genes, respectively. Here we evaluated the off-target editing profiles of clinical lead guide RNAs (gRNAs) that, when combined with adenine base editors (ABEs), correct the recurrent PAH P281L variant, PAH R408W variant, or ABCC6 R1164X...

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Literature Corpus work: 76852d84-6dc0-501c-a99a-9613f0bc45bd
DOI: 10.1101/2024.04.22.590531

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