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PU.1 inhibitor (Belfer Neurodegeneration Consortium)
- Development stage
- Preclinical
- Lead developer
- Neurodegeneration Consortium
- Modality
- Small Molecules
- Administration
- Intraperitoneal, Oral
01
Overview
PU.1 inhibitor (Belfer Neurodegeneration Consortium) is a small-molecule inhibitor of the transcription factor PU.1 (encoded by the *SPI1* gene), currently in preclinical development by the Belfer Neurodegeneration Consortium at MD Anderson Cancer Center. PU.1 is a master regulator of myeloid cell development and function, including microglia in the central nervous system. Genetic studies have identified *SPI1* as a risk locus for Alzheimer's disease, where elevated PU.1 activity drives a pro-inflammatory microglial phenotype that contributes to neurodegeneration. By selectively inhibiting PU.1, this therapeutic candidate aims to restore homeostatic microglial function and provide neuroprotection. Additionally, because PU.1 is a master regulator of hematopoiesis and is frequently dysregulated in myeloid leukemias, the inhibitor is also being explored as a potential treatment for leukemia, particularly acute myeloid leukemia (AML).
- Other names
- SPI1 inhibitorSPI-1 inhibitorSPI 1 inhibitorPU.1 small molecule inhibitor
02
Targets
SPI1 (PU.1)
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