释放基因转移与编辑治疗血液系统疾病的治愈潜力
Unlocking the curative potential of gene transfer and editing for hematopoietic disorders
造血干祖细胞基因治疗已为部分遗传性血液病和免疫缺陷病带来潜在治愈选择。本文回顾了近年来基因转移技术和基因编辑工具（如CRISPR-Cas系统）的改进，以及体外操作造血干祖细胞流程的优化。文章强调需要保证长期植入和克隆多样性，并采用毒性更低、不损伤基因组的预处理方案。作者同时讨论了临床转化中的关键限制，以及降低成本、让更多患者用上这类疗法的公平性问题。
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不需要生物学背景，多打比方
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摘要Abstract
Hematopoietic stem and progenitor cells (HSPCs) gene therapy may transform the therapeutic landscape for inherited hematological disorders and already offers potentially curative options for some diseases, including inborn errors of immunity and β-hemoglobinopathies. Its continued success relies on further refinement of gene transfer technologies, gene-editing tools such as clustered regularly interspaced short palindromic repeats (CRISPR) and associated systems (CRISPR-Cas), and optimized ex vivo HSPC manipulation protocols that ensure robust, long-term engraftment and clonal diversity with reduced-toxicity, nongenotoxic conditioning strategies. Here, we reviewed recent developments and refinements in gene transfer and editing technologies for HSPCs, while also discussing the critical limitations and hurdles to clinical translation, as recently presented at the New Investigator Committee Gene Therapy webinar. Future directions must prioritize integrating technological innovation with the development of equitable and simplified models to reduce costs and ensure that these life-saving cellular therapies reach patients worldwide.