Here the authors provide an overview of the topics they covered including insights into the novel applications of the technique as well as its strengths and limitations.
In this review, the authors will discuss the specific applications of gene-editing technologies in human HSPC, as informed by prior experience with gene addition strategies. HSPC are desirable but challenging targets; the specific mechanisms these cells evolved to protect themselves from DNA damage render them potentially more susceptible to oncogenesis, especially given their ability to self-renew and their long-term proliferative potential. They further review scientists’ experience with gene-editing technologies to date, focusing on strategies to move these techniques towards implementation in safe and effective clinical trials.
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Here the authors provide an overview of the topics they covered including insights into the novel applications of the technique as well as its strengths and limitations.