Why the study?
Manipulating genes in hematopoietic stem cells using conventional transgenesis approaches can be time-consuming, expensive, and challenging.
Population
Lethally-irradiated C57BL/6 mice transplanted with Cas9-expressing lineage-negative bone marrow cells
Comparison
Targeting gRNA and RFP vs non-targeting gRNA
Design
Preclinical experimental protocol
Follow-up
4 weeks after transplantation
Key result
Lentiviral CRISPR/Cas9-mediated genome editing achieved ~90% transduction of myeloid cells and ~70% of lymphoid cells at 4 weeks after transplantation in mice.
Authors
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Enables efficient HSC gene editing in mice; leaves open translation to human applications.
This protocol provides an efficient and economical method for high-throughput evaluation of hematopoiesis-regulatory genes in mouse disease models.
Sano et al. (2019) studied Hematopoietic cells in disease models. Lentiviral CRISPR/Cas9-mediated genome editing vs. Lentivirus expressing non-targeting gRNA was evaluated on Transduction efficiency of myeloid and lymphoid cells. Lentiviral CRISPR/Cas9-mediated genome editing achieved ~90% transduction of myeloid cells and ~70% of lymphoid cells at 4 weeks after transplantation in mice.
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