Improved CRISPR genome editing using small highly active and specific engineered RNA-guided nucleases
Small CRISPR Cas9 proteins have potential in gene therapies but generally have poor editing efficiency or specificity and often recognize sub-optimal PAM sequences. Here the authors characterise four small nucleases and use protein engineering to create effective in vivo editors.
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Nature Portfolio
2021
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oai:doaj.org-article:164ce27f7fcc420f8085cc9624163a4b2021-12-02T16:14:49ZImproved CRISPR genome editing using small highly active and specific engineered RNA-guided nucleases10.1038/s41467-021-24454-52041-1723https://doaj.org/article/164ce27f7fcc420f8085cc9624163a4b2021-07-01T00:00:00Zhttps://doi.org/10.1038/s41467-021-24454-5https://doaj.org/toc/2041-1723Small CRISPR Cas9 proteins have potential in gene therapies but generally have poor editing efficiency or specificity and often recognize sub-optimal PAM sequences. Here the authors characterise four small nucleases and use protein engineering to create effective in vivo editors.Moritz J. SchmidtAshish GuptaChristien BednarskiStefanie Gehrig-GianniniFlorian RichterChristian PitzlerMichael GamalindaChristina GalonskaRyo TakeuchiKui WangCaroline ReissKerstin DehneMichael J. LukasonAkiko NomaCindy Park-WindholMariacarmela AlloccaAlbena KantardzhievaShailendra SaneKarolina KosakowskaBrian CaffertyJan TebbeSarah J. SpencerScott MunzerChristopher J. ChengAbraham ScariaAndrew M. ScharenbergAndré CohnenWayne M. CocoNature PortfolioarticleScienceQENNature Communications, Vol 12, Iss 1, Pp 1-12 (2021) |
| institution |
DOAJ |
| collection |
DOAJ |
| language |
EN |
| topic |
Science Q |
| spellingShingle |
Science Q Moritz J. Schmidt Ashish Gupta Christien Bednarski Stefanie Gehrig-Giannini Florian Richter Christian Pitzler Michael Gamalinda Christina Galonska Ryo Takeuchi Kui Wang Caroline Reiss Kerstin Dehne Michael J. Lukason Akiko Noma Cindy Park-Windhol Mariacarmela Allocca Albena Kantardzhieva Shailendra Sane Karolina Kosakowska Brian Cafferty Jan Tebbe Sarah J. Spencer Scott Munzer Christopher J. Cheng Abraham Scaria Andrew M. Scharenberg André Cohnen Wayne M. Coco Improved CRISPR genome editing using small highly active and specific engineered RNA-guided nucleases |
| description |
Small CRISPR Cas9 proteins have potential in gene therapies but generally have poor editing efficiency or specificity and often recognize sub-optimal PAM sequences. Here the authors characterise four small nucleases and use protein engineering to create effective in vivo editors. |
| format |
article |
| author |
Moritz J. Schmidt Ashish Gupta Christien Bednarski Stefanie Gehrig-Giannini Florian Richter Christian Pitzler Michael Gamalinda Christina Galonska Ryo Takeuchi Kui Wang Caroline Reiss Kerstin Dehne Michael J. Lukason Akiko Noma Cindy Park-Windhol Mariacarmela Allocca Albena Kantardzhieva Shailendra Sane Karolina Kosakowska Brian Cafferty Jan Tebbe Sarah J. Spencer Scott Munzer Christopher J. Cheng Abraham Scaria Andrew M. Scharenberg André Cohnen Wayne M. Coco |
| author_facet |
Moritz J. Schmidt Ashish Gupta Christien Bednarski Stefanie Gehrig-Giannini Florian Richter Christian Pitzler Michael Gamalinda Christina Galonska Ryo Takeuchi Kui Wang Caroline Reiss Kerstin Dehne Michael J. Lukason Akiko Noma Cindy Park-Windhol Mariacarmela Allocca Albena Kantardzhieva Shailendra Sane Karolina Kosakowska Brian Cafferty Jan Tebbe Sarah J. Spencer Scott Munzer Christopher J. Cheng Abraham Scaria Andrew M. Scharenberg André Cohnen Wayne M. Coco |
| author_sort |
Moritz J. Schmidt |
| title |
Improved CRISPR genome editing using small highly active and specific engineered RNA-guided nucleases |
| title_short |
Improved CRISPR genome editing using small highly active and specific engineered RNA-guided nucleases |
| title_full |
Improved CRISPR genome editing using small highly active and specific engineered RNA-guided nucleases |
| title_fullStr |
Improved CRISPR genome editing using small highly active and specific engineered RNA-guided nucleases |
| title_full_unstemmed |
Improved CRISPR genome editing using small highly active and specific engineered RNA-guided nucleases |
| title_sort |
improved crispr genome editing using small highly active and specific engineered rna-guided nucleases |
| publisher |
Nature Portfolio |
| publishDate |
2021 |
| url |
https://doaj.org/article/164ce27f7fcc420f8085cc9624163a4b |
| work_keys_str_mv |
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