肿瘤细胞治疗研究
英文原题：Therapeutic applications of CRISPR/Cas9 mediated targeted gene editing in acute lymphoblastic leukemia: current perspectives, future challenges, and clinical implications.
Therapeutic applications of CRISPR/Cas9 mediated targeted gene editing in acute lymphoblastic leukemia: current perspectives, future challenges, and clinical implications.
急性淋巴细胞白血病（ALL）是儿童人群中最主要的血液系统恶性肿瘤，起源于骨髓内的 B 细胞或 T 细胞前体。
急性淋巴细胞白血病（ALL）是儿童人群中最常见的血液系统恶性肿瘤，起源于骨髓中的B细胞或T细胞前体。该疾病在分子层面和临床表现方面均具有高度异质性。遗传和获得性基因改变之间复杂的相互作用参与疾病发病机制，常导致白血病发生过程中关键细胞功能紊乱。CRISPR/Cas9作为基因编辑工具的出现革新了生物学研究，凸显了其修饰癌症相关特定基因组位点的潜力。对ALL分子改变的深入认识推动治疗策略取得重大进展。本综述考察CRISPR/Cas9作为识别遗传靶点工具的应用，以改进治疗、克服耐药并促进基于CAR-T细胞的免疫治疗；此外，我们讨论CRISPR/Cas9在ALL中的应用挑战和未来前景。
Acute Lymphoblastic Leukemia (ALL) is the predominant hematological malignancy in pediatric populations, originating from B- or T-cell precursors within the bone marrow. The disease exhibits a high degree of heterogeneity, both at the molecular level and in terms of clinical presentation. A complex interplay between inherited and acquired genetic alterations contributes to disease pathogenesis, often resulting in the disruption of cellular functions integral to the leukemogenic process. The advent of CRISPR/Cas9 as a gene editing tool has revolutionized biological research, underscoring its potential to modify specific genomic loci implicated in cancer. Enhanced understanding of molecular alterations in ALL has facilitated significant advancements in therapeutic strategies. In this review, we scrutinize the application of CRISPR/Cas9 as a tool for identifying genetic targets to improve therapy, circumvent drug resistance, and facilitate CAR-T cell-based immunotherapy. Additionally, we discuss the challenges and future prospects of CRISPR/Cas9 applications in ALL.
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