Optimizing Gene Editing with a Combination of mRNA and AAV
In the past few years there have been major advances towards fast, efficient, and precise gene editing. However, there are still several hurdles to overcome, particularly for certain cell types. Hematopoietic stem and progenitor cells (HSPCs) are notoriously hard to edit, especially in cases where the donor DNA template is large, such as when the goal is to replace an entire gene. And even more challenging, is to target the most primitive CD34+CD133+CD90+ HPSC cell population, which contains the repopulating hematopoietic stem cells necessary for long-term engraftment and differentiation.
In a recent paper published in Nature Biotechnology, researchers led by Michael Holmes and Paula Cannon sought to make this process more efficient through the use of...
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