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Selector adeno-associated viral vectors facilitate on-target precise genome editing and purge off-target chromosomal insertions - PubMed

6 hours ago
  • #AAV Vectors
  • #Precision Genome Editing
  • #Off-Target Purging
  • Selector adeno-associated viral (AAV) vectors with marker-free selectable sequences enable enrichment for cells with precise on-target edits at endogenous alleles.
  • These vectors introduce ATP1A1 polymorphisms conferring resistance to ouabain, achieving high frequencies of accurately edited cell populations.
  • Selector AAV vectors effectively purge heterogeneous off-target DNA insertions that arise from conventional AAV-based genome editing methods.
  • The approach mitigates issues like off-target chromosomal insertions via nonhomologous end joining and imprecise on-target edits from end-joining pathways.