Key result
The combination of CRISPR/Cas9 RNP complex and recombinant AAV6 vectors resulted in successful transgene delivery (6.85% mCherry+ cells) into the AAVS1 safe harbor site of human iPSCs.
Why the study?
Precise transgene insertion in hiPSCs represents a significant challenge because viral transduction causes random integration and non-viral strategies suffer from low transfection efficiency.
Population
Human induced pluripotent stem cells
Design
Protocol description
Authors
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Facilitates precise gene editing in human iPSCs; leaves open clinical translation pending in vivo validation.
Absolute Event Rate: 6.85% vs 0%
The combination of CRISPR/Cas9 RNP complex and recombinant AAV6 vectors enables precise and efficient transgene knock-in at the AAVS1 safe harbor locus in human iPSCs.
Chupradit et al. (2022) studied In vitro human induced pluripotent stem cells (hiPSCs). CRISPR/Cas9 RNP complex combined with recombinant AAV6 donor vectors vs. RNP + pAAV donor plasmid or AAV6 alone was evaluated on Percentage of mCherry+ cells (transgene insertion). The combination of CRISPR/Cas9 RNP complex and recombinant AAV6 vectors resulted in successful transgene delivery (6.85% mCherry+ cells) into the AAVS1 safe harbor site of human iPSCs.
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