Details

Autor(en) / Beteiligte

• Zafra, Maria Paz
• Schatoff, Emma M
• Katti, Alyna
• Foronda, Miguel
• Breinig, Marco
• Schweitzer, Anabel Y
• Simon, Amber
• Han, Teng
• Goswami, Sukanya
• Montgomery, Emma
• Thibado, Jordana
• Kastenhuber, Edward R
• Sánchez-Rivera, Francisco J
• Shi, Junwei
• Vakoc, Christopher R
• Lowe, Scott W
• Tschaharganeh, Darjus F
• Dow, Lukas E

Titel

Optimized base editors enable efficient editing in cells, organoids and mice

Ist Teil von

• Nature biotechnology, 2018-10, Vol.36 (9), p.888-893

Ort / Verlag

United States: Nature Publishing Group

Erscheinungsjahr

2018

Link zum Volltext

Quelle

MEDLINE

Beschreibungen/Notizen

• CRISPR base editing enables the creation of targeted single-base conversions without generating double-stranded breaks. However, the efficiency of current base editors is very low in many cell types. We reengineered the sequences of BE3, BE4Gam, and xBE3 by codon optimization and incorporation of additional nuclear-localization sequences. Our collection of optimized constitutive and inducible base-editing vector systems dramatically improves the efficiency by which single-nucleotide variants can be created. The reengineered base editors enable target modification in a wide range of mouse and human cell lines, and intestinal organoids. We also show that the optimized base editors mediate efficient in vivo somatic editing in the liver in adult mice.