Targeted nonviral delivery of genome editors in vivo

Tsuchida, Connor A.; Wasko, Kevin M.; Hamilton, Jennifer R.; Doudna, Jennifer A.

doi:10.1073/pnas.2307796121

Title: Targeted nonviral delivery of genome editors in vivo

Journal Article · 04 March 2024 · Proceedings of the National Academy of Sciences of the United States of America

DOI: https://doi.org/10.1073/pnas.2307796121 · OSTI ID:2470986

^[1]; Wasko, Kevin M. ^[1];

^[1];

^[2]

University of California, Berkeley, CA (United States)
University of California, Berkeley, CA (United States); Lawrence Berkeley National Laboratory (LBNL), Berkeley, CA (United States); University of California, San Francisco, CA (United States)

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.

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Research Organization:: Lawrence Berkeley National Laboratory (LBNL), Berkeley, CA (United States)

Sponsoring Organization:: USDOE

Grant/Contract Number:: AC02-05CH11231

OSTI ID:: 2470986

Journal Information:: Proceedings of the National Academy of Sciences of the United States of America, Journal Name: Proceedings of the National Academy of Sciences of the United States of America Journal Issue: 11 Vol. 121; ISSN 0027-8424

Publisher:: National Academy of SciencesCopyright Statement

Country of Publication:: United States

Language:: English

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Delivering SaCas9 mRNA by lentivirus-like bionanoparticles for transient expression and efficient genome editing Lu, Baisong; Javidi-Parsijani, Parisa; Makani, Vishruti Nucleic Acids Research, Vol. 47, Issue 8 https://doi.org/10.1093/nar/gkz093	journal	February 2019
Delivering Cas9/sgRNA ribonucleoprotein (RNP) by lentiviral capsid-based bionanoparticles for efficient ‘hit-and-run’ genome editing Lyu, Pin; Javidi-Parsijani, Parisa; Atala, Anthony Nucleic Acids Research, Vol. 47, Issue 17 https://doi.org/10.1093/nar/gkz605	journal	July 2019
Antibody-mediated delivery of CRISPR-Cas9 ribonucleoproteins in human cells Ubiparipovic, Stephanie; Christ, Daniel; Rouet, Romain Protein Engineering, Design and Selection, Vol. 35 https://doi.org/10.1093/protein/gzac011	journal	February 2022
Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins Kim, S.; Kim, D.; Cho, S. W. Genome Research, Vol. 24, Issue 6 https://doi.org/10.1101/gr.171322.113	journal	April 2014
Genome surgery using Cas9 ribonucleoproteins for the treatment of age-related macular degeneration Kim, Kyoungmi; Park, Sung Wook; Kim, Jin Hyoung Genome Research, Vol. 27, Issue 3 https://doi.org/10.1101/gr.219089.116	journal	February 2017
Codelivery of CRISPR-Cas9 and chlorin e6 for spatially controlled tumor-specific gene editing with synergistic drug effects Deng, Shaohui; Li, Xiaoxia; Liu, Shuang Science Advances, Vol. 6, Issue 29 https://doi.org/10.1126/sciadv.abb4005	journal	July 2020
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Blood-brain barrier–penetrating single CRISPR-Cas9 nanocapsules for effective and safe glioblastoma gene therapy Zou, Yan; Sun, Xinhong; Yang, Qingshan Science Advances, Vol. 8, Issue 16 https://doi.org/10.1126/sciadv.abm8011	journal	April 2022
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CRISPR-engineered T cells in patients with refractory cancer Stadtmauer, Edward A.; Fraietta, Joseph A.; Davis, Megan M. Science, Vol. 367, Issue 6481 https://doi.org/10.1126/science.aba7365	journal	February 2020
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Baboon Envelope Pseudotyped “Nanoblades” Carrying Cas9/gRNA Complexes Allow Efficient Genome Editing in Human T, B, and CD34+ Cells and Knock-in of AAV6-Encoded Donor DNA in CD34+ Cells Gutierrez-Guerrero, Alejandra; Abrey Recalde, Maria Jimena; Mangeot, Philippe E. Frontiers in Genome Editing, Vol. 3 https://doi.org/10.3389/fgeed.2021.604371	journal	February 2021
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Pseudotyping Lentiviral Vectors: When the Clothes Make the Virus Duvergé, Alexis; Negroni, Matteo Viruses, Vol. 12, Issue 11 https://doi.org/10.3390/v12111311	journal	November 2020
Targeted genome editing by lentiviral protein transduction of zinc-finger and TAL-effector nucleases Cai, Yujia; Bak, Rasmus O.; Mikkelsen, Jacob Giehm eLife, Vol. 3 https://doi.org/10.7554/eLife.01911	journal	April 2014

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