AI Article Synopsis
- Researchers developed a CRISPR/Cas9/AAV-based system to introduce and repair mutations in mouse hematopoietic stem and progenitor cells (HSPCs), which can lead to severe blood disorders.
- The protocol allows for efficient gene knock-in while ensuring that the cells can still successfully engraft.
- This method enables the examination of hematopoietic disease mutations without altering the germline, providing a reliable way to study these genetic changes in a laboratory setting.
Article Abstract
Mutations that accumulate in self-renewing hematopoietic stem and progenitor cells (HSPCs) can cause severe blood disorders. To model such disorders in mice, we developed a CRISPR/Cas9/adeno-associated virus (AAV)-based system to knock in and repair genes by homologous recombination in mouse HSPCs. Here, we provide a step-by-step protocol to achieve high efficiency of gene knockin in mouse HSPCs, while maintaining engraftment capacity. This approach enables the functional study of hematopoietic disease mutations , without requiring germline mutagenesis. For complete details on the use and execution of this protocol, please refer to Tran et al. (2019).
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| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7357675 | PMC |
| http://dx.doi.org/10.1016/j.xpro.2020.100028 | DOI Listing |
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