Targeted exon skipping with AAV-mediated split adenine base editors
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DOI: 10.1038/s41421-019-0109-7
Abstract: Techniques for exclusion of exons from mature transcripts have been applied as gene therapies for treating many different diseases. Since exon skipping has been traditionally accomplished using technologies that have a transient effect, it is… read more here.
Keywords: targeted exon; base editors; adenine base; base ... See more keywords