Improved CRISPR genome editing using small highly active and specific engineered RNA-guided nucleases

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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Detalles Bibliográficos
Autores principales: 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
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2021
Materias:
Science
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Acceso en línea:https://doaj.org/article/164ce27f7fcc420f8085cc9624163a4b
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Descripción
Sumario: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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