Targeted nonviral delivery of genome editors in vivo

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 e...

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Bibliographic Details
Published in:Proceedings of the National Academy of Sciences - PNAS 2024-03, Vol.121 (11), p.e2307796121-e2307796121
Main Authors: Tsuchida, Connor A, Wasko, Kevin M, Hamilton, Jennifer R, Doudna, Jennifer A
Format: Article
Language:English
Subjects:
Biological Sciences
Commerce
CRISPR
Editing
Gene Editing
Gene therapy
Genetic Therapy
Genomes
mRNA
Ribonucleoproteins
RNA, Messenger
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Summary: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.
ISSN:0027-8424
1091-6490
DOI:10.1073/pnas.2307796121