Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery

Authors:
Lin S, Staahl BT, Alla RK, Doudna JA.
In:
Source: eLife
Publication Date: (2014)
Issue: 3: e04766
Research Area:
Cancer Research/Cell Biology
Stem Cells
Basic Research
Cells used in publication:
Fibroblast, dermal (NHDF-Neo), human neonatal
Species: human
Tissue Origin: dermal
293T
Species: human
Tissue Origin: kidney
Platform:
4D-Nucleofector® 96-well Systems
Experiment
Cas9 RNP assembly and nucleofection: Cas9 RNP was prepared immediately before experiment by incubating with sgRNA at 1:1.2 molar ratio in 20 mM HEPES (pH 7.5), 150 mM KCl, 1 mM MgCl2, 10% glycerol and 1 mM TCEP at 37°C for 10 min. HDR template was then added to the RNP mixture. Cells were dissociated by 0.05% trypsin, spun down by centrifugation at 400×g for 3 min, and washed once with DPBS. Nucleofection of HEK293T cells was performed using Lonza (Allendale, NJ) SF cell- kits and program CM130 in an Amaxa 96-well Shuttle system. The human neoFB were transfected with Lonza P2 kit and program CA137. The hES cells were transfected with P3 primary cell kit and program CB150. Each nucleofection reaction consisted of approximately 2 × 105 cells in 20 µl of nucleofection reagent and mixed with 10 µl of RNP:DNA. After electroporation, 100 µl of growth media was added to the well to transfer the cells to tissue culture plates. The cells were incubated at 37°C for 24 hr...
Abstract
The CRISPR/Cas9 system is a robust genome editing technology that works in human cells, animals and plants based on the RNA-programmed DNA cleaving activity of the Cas9 enzyme. Building on previous work (Jinek et al., 2013), we show here that new genetic information can be introduced site-specifically and with high efficiency by homology-directed repair (HDR) of Cas9-induced site-specific double-strand DNA breaks using timed delivery of Cas9-guide RNA ribonucleoprotein (RNP) complexes. Cas9 RNP-mediated HDR in HEK293T, human primary neonatal fibroblast and human embryonic stem cells was increased dramatically relative to experiments in unsynchronized cells, with rates of HDR up to 38% observed in HEK293T cells. Sequencing of on- and potential off-target sites showed that editing occurred with high fidelity, while cell mortality was minimized. This approach provides a simple and highly effective strategy for enhancing site-specific genome engineering in both transformed and primary human cells.