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Targeted nonviral delivery of genome editors in vivo.

Connor A TsuchidaKevin M WaskoJennifer R HamiltonJennifer A Doudna
Published in: Proceedings of the National Academy of Sciences of the United States of America (2024)
Cell-type-specific in vivo delivery of genome editing molecules is the next breakthrough that will drive biological discovery and transform the field of cell and gene therapy. Here, we discuss recent advances in the delivery of CRISPR-Cas genome editors either as preassembled ribonucleoproteins or encoded in mRNA. Both strategies avoid pitfalls of viral vector-mediated delivery and offer advantages including transient editor lifetime and potentially streamlined manufacturing capability that are already proving valuable for clinical use. We review current applications and future opportunities of these emerging delivery approaches that could make genome editing more efficacious and accessible in the future.
Keyphrases
  • crispr cas
  • genome editing
  • gene therapy
  • sars cov
  • current status
  • stem cells
  • genome wide
  • drug delivery
  • high throughput
  • bone marrow