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Updated: May 31, 2026

Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
Published on: September 18, 2013
[Therapeutic effect of focal adhesion kinase gene silence on leukemia]
Lü-Hong Xu1, Jian-Pei Fang, Wen-Jun Weng
1Department of Pediatrics, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, Guangzhou 510120, Guangdong Province, China.
Abstract:
This study was aimed to investigate the effects of focal adhesion kinase (FAK) gene silence on leukemia cell growth, leukemogenesis and efficacy of chemotherapy drug. Vector containing lentiviral-FAK-shRNA was constructed and transfected into BCR/ABL-BaF3 leukemic cells, the cell growth and apoptosis were detected in vitro. The effect of FAK shRNA on leukemogenesis was studied in a murine model with leukemia. The apoptosis of leukemia cells and survival of leukemic mice treated by FAK shRNA combined with drug STI571 were monitored. The results showed that FAK gene expression was knocked down by lentiviral-FAK-shRNA. FAK gene silencing inhibited leukemia cell growth in vitro. The apoptosis test results showed that the percentages of Annexin V(+) cells in vector control group and FAK shRNA group were (3.46 ± 0.56)% and (7.3 ± 0.79)%, respectively, and the difference was statistically significant (p < 0.05). The mice in vector control group died at day 21 to 27, while the mice in FAK shRNA group died between day 52 and 60, and the difference was statistically significant (p < 0.05). Moreover, FAK gene silence combined with drug STI571 could enhance the apoptosis of leukemia cells and prolong survival time of leukemic mice. It is concluded that FAK gene silence inhibits leukemogenesis and promotes efficacy of chemotherapy drug on leukemia cells, indicating FAK gene silence may be considered as a new therapeutic strategy for leukemia.
Insights
Focal adhesion kinase (FAK) gene silencing inhibits leukemia cell growth and progression. This approach enhances chemotherapy effectiveness, suggesting FAK gene silencing as a potential new leukemia therapy.
Area of Science:
- Molecular Biology
- Cancer Research
- Gene Therapy
Context:
- Leukemia remains a significant health challenge, necessitating novel therapeutic strategies.
- Focal adhesion kinase (FAK) plays a role in cell signaling pathways crucial for cancer progression.
- Targeting FAK offers a potential avenue for leukemia treatment.
Purpose:
- To investigate the impact of focal adhesion kinase (FAK) gene silencing on leukemia.
- To evaluate the efficacy of FAK gene silencing in combination with chemotherapy (STI571).
- To assess the effects on leukemia cell growth, apoptosis, and leukemogenesis in vitro and in vivo.
Summary:
- Lentiviral-FAK-shRNA effectively silenced FAK gene expression in BCR/ABL-BaF3 cells.
- FAK gene silencing significantly inhibited leukemia cell proliferation and induced apoptosis in vitro.
- In a murine leukemia model, FAK shRNA treatment prolonged survival and enhanced the apoptotic effects of STI571.
Impact:
- FAK gene silencing demonstrates potential as a novel therapeutic strategy for leukemia.
- Combining FAK inhibition with chemotherapy may improve treatment outcomes.
- This research provides a foundation for developing targeted therapies against FAK in leukemia.
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