A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing
The development of clinically viable delivery methods presents one of the greatest challenges in the therapeutic application of CRISPR/Cas9 mediated genome editing. Here, we report the development of a lipid nanoparticle (LNP)-mediated delivery system that, with a single administration, enabled sign...
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| Published in | Cell reports (Cambridge) Vol. 22; no. 9; pp. 2227 - 2235 |
|---|---|
| Main Authors | , , , , , , , , , , , , , , , , , , , , , , |
| Format | Journal Article |
| Language | English |
| Published |
United States
Elsevier Inc
27.02.2018
Elsevier |
| Subjects | |
| Online Access | Get full text |
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| Abstract | The development of clinically viable delivery methods presents one of the greatest challenges in the therapeutic application of CRISPR/Cas9 mediated genome editing. Here, we report the development of a lipid nanoparticle (LNP)-mediated delivery system that, with a single administration, enabled significant editing of the mouse transthyretin (Ttr) gene in the liver, with a >97% reduction in serum protein levels that persisted for at least 12 months. These results were achieved with an LNP delivery system that was biodegradable and well tolerated. The LNP delivery system was combined with a sgRNA having a chemical modification pattern that was important for high levels of in vivo activity. The formulation was similarly effective in a rat model. Our work demonstrates that this LNP system can deliver CRISPR/Cas9 components to achieve clinically relevant levels of in vivo genome editing with a concomitant reduction of TTR serum protein, highlighting the potential of this system as an effective genome editing platform.
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•LNP delivery achieves >97% target protein knockdown for at least 12 months•Editing level is cumulative following multiple LNP doses•A sgRNA chemical modification pattern was critical for high levels of in vivo activity•Biodegradable lipid and CRISPR/Cas9 components are transient and well tolerated
Finn et al. describe the development of a transient, biodegradable LNP-based CRISPR/Cas9 delivery system that achieves >97% knockdown of serum TTR levels following a single administration. Editing levels were stable for 12 months, despite the transient nature of the delivery system and the editing components. |
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| AbstractList | The development of clinically viable delivery methods presents one of the greatest challenges in the therapeutic application of CRISPR/Cas9 mediated genome editing. Here, we report the development of a lipid nanoparticle (LNP)-mediated delivery system that, with a single administration, enabled significant editing of the mouse transthyretin (Ttr) gene in the liver, with a >97% reduction in serum protein levels that persisted for at least 12 months. These results were achieved with an LNP delivery system that was biodegradable and well tolerated. The LNP delivery system was combined with a sgRNA having a chemical modification pattern that was important for high levels of in vivo activity. The formulation was similarly effective in a rat model. Our work demonstrates that this LNP system can deliver CRISPR/Cas9 components to achieve clinically relevant levels of in vivo genome editing with a concomitant reduction of TTR serum protein, highlighting the potential of this system as an effective genome editing platform. : Finn et al. describe the development of a transient, biodegradable LNP-based CRISPR/Cas9 delivery system that achieves >97% knockdown of serum TTR levels following a single administration. Editing levels were stable for 12 months, despite the transient nature of the delivery system and the editing components. Keywords: CRISPR, Cas9, genome editing, LNP, lipid nanoparticle, TTR, CRISPR/Cas9, liver delivery, gene therapy, sgRNA The development of clinically viable delivery methods presents one of the greatest challenges in the therapeutic application of CRISPR/Cas9 mediated genome editing. Here, we report the development of a lipid nanoparticle (LNP)-mediated delivery system that, with a single administration, enabled significant editing of the mouse transthyretin (Ttr) gene in the liver, with a >97% reduction in serum protein levels that persisted for at least 12 months. These results were achieved with an LNP delivery system that was biodegradable and well tolerated. The LNP delivery system was combined with a sgRNA having a chemical modification pattern that was important for high levels of in vivo activity. The formulation was similarly effective in a rat model. Our work demonstrates that this LNP system can deliver CRISPR/Cas9 components to achieve clinically relevant levels of in vivo genome editing with a concomitant reduction of TTR serum protein, highlighting the potential of this system as an effective genome editing platform.The development of clinically viable delivery methods presents one of the greatest challenges in the therapeutic application of CRISPR/Cas9 mediated genome editing. Here, we report the development of a lipid nanoparticle (LNP)-mediated delivery system that, with a single administration, enabled significant editing of the mouse transthyretin (Ttr) gene in the liver, with a >97% reduction in serum protein levels that persisted for at least 12 months. These results were achieved with an LNP delivery system that was biodegradable and well tolerated. The LNP delivery system was combined with a sgRNA having a chemical modification pattern that was important for high levels of in vivo activity. The formulation was similarly effective in a rat model. Our work demonstrates that this LNP system can deliver CRISPR/Cas9 components to achieve clinically relevant levels of in vivo genome editing with a concomitant reduction of TTR serum protein, highlighting the potential of this system as an effective genome editing platform. The development of clinically viable delivery methods presents one of the greatest challenges in the therapeutic application of CRISPR/Cas9 mediated genome editing. Here, we report the development of a lipid nanoparticle (LNP)-mediated delivery system that, with a single administration, enabled significant editing of the mouse transthyretin (Ttr) gene in the liver, with a >97% reduction in serum protein levels that persisted for at least 12 months. These results were achieved with an LNP delivery system that was biodegradable and well tolerated. The LNP delivery system was combined with a sgRNA having a chemical modification pattern that was important for high levels of in vivo activity. The formulation was similarly effective in a rat model. Our work demonstrates that this LNP system can deliver CRISPR/Cas9 components to achieve clinically relevant levels of in vivo genome editing with a concomitant reduction of TTR serum protein, highlighting the potential of this system as an effective genome editing platform. The development of clinically viable delivery methods presents one of the greatest challenges in the therapeutic application of CRISPR/Cas9 mediated genome editing. Here, we report the development of a lipid nanoparticle (LNP)-mediated delivery system that, with a single administration, enabled significant editing of the mouse transthyretin (Ttr) gene in the liver, with a >97% reduction in serum protein levels that persisted for at least 12 months. These results were achieved with an LNP delivery system that was biodegradable and well tolerated. The LNP delivery system was combined with a sgRNA having a chemical modification pattern that was important for high levels of in vivo activity. The formulation was similarly effective in a rat model. Our work demonstrates that this LNP system can deliver CRISPR/Cas9 components to achieve clinically relevant levels of in vivo genome editing with a concomitant reduction of TTR serum protein, highlighting the potential of this system as an effective genome editing platform. [Display omitted] •LNP delivery achieves >97% target protein knockdown for at least 12 months•Editing level is cumulative following multiple LNP doses•A sgRNA chemical modification pattern was critical for high levels of in vivo activity•Biodegradable lipid and CRISPR/Cas9 components are transient and well tolerated Finn et al. describe the development of a transient, biodegradable LNP-based CRISPR/Cas9 delivery system that achieves >97% knockdown of serum TTR levels following a single administration. Editing levels were stable for 12 months, despite the transient nature of the delivery system and the editing components. |
| Author | Smith, Amy Rhoden Patel, Mihir C. Shaw, Lucinda Morrissey, David V. van Heteren, Jane Harrington, William F. Pink, Melissa Rohde, Ellen Strapps, Walter R. Shah, Aalok Finn, Jonathan D. Growe, Jacqueline Seitzer, Jessica Wood, Kristy M. Shah, Ruchi R. Dirstine, Tanner Youniss, Madeleine R. Salomon, William E. Lescarbeau, Reynald Ling, Dandan Dombrowski, Christian Chang, Yong Ciullo, Corey |
| Author_xml | – sequence: 1 givenname: Jonathan D. surname: Finn fullname: Finn, Jonathan D. organization: Intellia Therapeutics, Cambridge, MA 02139, USA – sequence: 2 givenname: Amy Rhoden surname: Smith fullname: Smith, Amy Rhoden organization: Intellia Therapeutics, Cambridge, MA 02139, USA – sequence: 3 givenname: Mihir C. surname: Patel fullname: Patel, Mihir C. organization: Intellia Therapeutics, Cambridge, MA 02139, USA – sequence: 4 givenname: Lucinda surname: Shaw fullname: Shaw, Lucinda organization: Intellia Therapeutics, Cambridge, MA 02139, USA – sequence: 5 givenname: Madeleine R. surname: Youniss fullname: Youniss, Madeleine R. organization: Intellia Therapeutics, Cambridge, MA 02139, USA – sequence: 6 givenname: Jane surname: van Heteren fullname: van Heteren, Jane organization: Intellia Therapeutics, Cambridge, MA 02139, USA – sequence: 7 givenname: Tanner surname: Dirstine fullname: Dirstine, Tanner organization: Intellia Therapeutics, Cambridge, MA 02139, USA – sequence: 8 givenname: Corey surname: Ciullo fullname: Ciullo, Corey organization: Intellia Therapeutics, Cambridge, MA 02139, USA – sequence: 9 givenname: Reynald surname: Lescarbeau fullname: Lescarbeau, Reynald organization: Intellia Therapeutics, Cambridge, MA 02139, USA – sequence: 10 givenname: Jessica surname: Seitzer fullname: Seitzer, Jessica organization: Intellia Therapeutics, Cambridge, MA 02139, USA – sequence: 11 givenname: Ruchi R. surname: Shah fullname: Shah, Ruchi R. organization: Intellia Therapeutics, Cambridge, MA 02139, USA – sequence: 12 givenname: Aalok surname: Shah fullname: Shah, Aalok organization: Intellia Therapeutics, Cambridge, MA 02139, USA – sequence: 13 givenname: Dandan surname: Ling fullname: Ling, Dandan organization: Intellia Therapeutics, Cambridge, MA 02139, USA – sequence: 14 givenname: Jacqueline surname: Growe fullname: Growe, Jacqueline organization: Intellia Therapeutics, Cambridge, MA 02139, USA – sequence: 15 givenname: Melissa surname: Pink fullname: Pink, Melissa organization: Intellia Therapeutics, Cambridge, MA 02139, USA – sequence: 16 givenname: Ellen surname: Rohde fullname: Rohde, Ellen organization: Intellia Therapeutics, Cambridge, MA 02139, USA – sequence: 17 givenname: Kristy M. surname: Wood fullname: Wood, Kristy M. organization: Intellia Therapeutics, Cambridge, MA 02139, USA – sequence: 18 givenname: William E. surname: Salomon fullname: Salomon, William E. organization: Intellia Therapeutics, Cambridge, MA 02139, USA – sequence: 19 givenname: William F. surname: Harrington fullname: Harrington, William F. organization: Intellia Therapeutics, Cambridge, MA 02139, USA – sequence: 20 givenname: Christian surname: Dombrowski fullname: Dombrowski, Christian organization: Intellia Therapeutics, Cambridge, MA 02139, USA – sequence: 21 givenname: Walter R. surname: Strapps fullname: Strapps, Walter R. organization: Intellia Therapeutics, Cambridge, MA 02139, USA – sequence: 22 givenname: Yong surname: Chang fullname: Chang, Yong organization: Intellia Therapeutics, Cambridge, MA 02139, USA – sequence: 23 givenname: David V. surname: Morrissey fullname: Morrissey, David V. email: davidm@intelliatx.com organization: Intellia Therapeutics, Cambridge, MA 02139, USA |
| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/29490262$$D View this record in MEDLINE/PubMed |
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| Publisher_xml | – name: Elsevier Inc – name: Elsevier |
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| SubjectTerms | Animals Base Sequence Cas9 CRISPR CRISPR-Associated Protein 9 - metabolism CRISPR-Cas Systems - genetics CRISPR/Cas9 Gene Editing gene therapy Gene Transfer Techniques genome editing lipid nanoparticle Lipids - chemistry Liver - metabolism liver delivery LNP Mice Nanoparticles - administration & dosage Nanoparticles - chemistry Rats RNA, Guide, CRISPR-Cas Systems - chemistry RNA, Guide, CRISPR-Cas Systems - genetics sgRNA TTR |
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| Title | A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing |
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