Target intelligence / Profile preview

Abnormal spindle-like microcephaly-associated protein (ASPM)

Target
ASPM
Molecular classification
Other (Mitotic spindle assembly protein), Spindle assembly factor, Microtubule-associated protein
01

Overview

Abnormal spindle-like microcephaly-associated protein (ASPM) is a large, conserved protein required for proper mitotic spindle function during cell division, particularly in neural progenitor cells[1][2][5]. It localizes to spindle poles and the midbody, with a key role in regulating spindle orientation, astral microtubule density, and ensuring balanced symmetric cell divisions[3][5]. In humans, loss-of-function mutations in the ASPM gene are the most common cause of autosomal recessive primary microcephaly, a disorder marked by reduced brain size with relatively intact architecture due to defective neurogenesis and proliferation[1][2][3]. ASPM’s function is also implicated in the proliferation of various tumors; high expression correlates with increased tumor growth and worse clinical outcomes in several CNS cancers[2][4]. While not a classical drug target (such as a kinase or receptor), ASPM's roles in cancer biology and developmental disease make it a putative target of therapeutic and research interest.

Other names
abnormal spindle protein homologAsp homologMCPH5Calmbp1ASPFLJ10517FLJ10549abnormal spindle-like microcephaly-associated proteinabnormal spindle microtubule assemblyasp (abnormal spindle) homologmicrocephaly associated
02

Biological functions

Mitotic spindle organizationCell division (especially symmetric division in neural progenitors)Cell proliferationDNA repair and replication stress responseRegulation of neurogenesisSpindle orientation
03

Disease associations

Primary microcephaly (autosomal recessive primary microcephaly, MCPH)Cancer (proliferation and poor prognosis in various tumors, including medulloblastoma and glioblastoma)Other developmental disorders linked to abnormal cell division
04

Safety considerations

Tumorigenic potential when overexpressed (potential oncogene/high levels correlate with poor outcomes in CNS tumors)Loss-of-function mutations cause microcephaly and major CNS developmental disorders
05

Biomarkers

ASPM overexpression as a biomarker of poor prognosis in certain cancers (e.g., medulloblastoma, ependymoma, glioblastoma)

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