Mab immunotherapies for myeloid leukemia patients with germline or somatic RUNX1 mutations.

这一建议提供了初步的证据，并建议证实两个细胞表面分子CD11a(ITGAL)和IL3RA(CD123)是HSCs中RUNX1的直接(可能是抑制)靶点，并且在RUNX1突变的AML中调节失调。针对这两种表面分子的单抗疗法已经针对不同的疾病通过了不同的临床试验阶段。我们计划在临床前模型中证明这些抗体对RUNX1阳性AML有效，然后进行临床试验。

This proposal presents preliminary evidence and proposes to confirm that 2 cell surface molecules, CD11a (ITGAL) and IL3RA (CD123) are direct (probably repression) targets of RUNX1 in HSCs, and are dysregulated in RUNX1 mutated AML. Monoclonal antibody therapies that target these two surface molecules have already passed different clinical trial phases for different diseases. We plan to show these antibodies are effective in RUNX1 positive AML in preclinical models and then clinical trials.