Due to pioneering in vitro investigations on gene modification, gene engineering platforms have incredibly improved to a safer and more powerful tool for the treatment of multiple blood and immune disorders. Likewise, several clinical trials have been initiated combining autologous hematopoietic stem cell transplantation (auto-HSCT) with gene therapy (GT) tools. As several GT modalities such as lentivirus and gene editing tools have a long developmental path ahead to diminish its negative side effects, it is hard to decide which modality is optimal for treating a specific disease. Gene transfer by lentiviruses is the platform of choice for loss-of-mutation diseases, whereas gene correction/addition or gene disruption by gene editing tools, mainly CRISPR/Cas9, is likely to be more efficient in diseases where tight regulation is needed. Therefore, in this review, we compiled pertinent information about lentiviral gene transfer and CRISPR/Cas9 gene editing, their evolution to a safer platform for HSCT, and their applications on other types of gene disorders based on the etiology of the disease and cell fitness.

由于对基因修饰进行了体外研究的开创性研究，基因工程平台已得到不可思议的改进，使其成为治疗多种血液和免疫疾病的更安全，更强大的工具。同样，已经启动了一些将自体造血干细胞移植（auto-HSCT）与基因治疗（GT）工具相结合的临床试验。由于慢病毒和基因编辑工具等几种GT方式在消除其负面副作用方面还有很长的发展道路，因此很难确定哪种方式最适合治疗特定疾病。慢病毒的基因转移是突变丢失疾病的选择平台，而基因编辑工具（主要是CRISPR / Cas9）进行的基因校正/添加或基因破坏可能在需要严格调控的疾病中更为有效。因此，在这篇评论中，