Two recent papers (one by Román-Rodríguez et al., 2019 in this issue of Cell Stem Cell) highlight how the power of biological selection on hematopoietic stem cell fitness can facilitate gene therapies for Fanconi Anemia. A clinical trial using lentiviral gene replacement and a proof-of-concept targeted genome editing study show robust engraftment and expansion of gene-corrected cells at levels reaching therapeutic relevance.

中文翻译：

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基因校正的HSPC的体内选择促进了罕见干细胞疾病的基因治疗。

最近的两篇论文（由Román-Rodríguez等人撰写，2019年在本期《细胞干细胞》中发表）强调了生物学选择对造血干细胞适应性的功效如何促进Fanconi贫血的基因治疗。一项使用慢病毒基因替代和概念验证的靶向基因组编辑研究的临床试验显示，经过基因校正的细胞能够稳固地植入并扩展至达到治疗相关性的水平。