Nasu-Hakola disease

Summary

Nasu-Hakola disease is a rare autosomal recessive leukodystrophy characterized by early-onset dementia and systemic bone cysts, representing the extreme consequence of total microglial and osteoclast failure.

Microglial Relevance

Caused directly by bi-allelic loss-of-function variants in either TREM2 or TYROBP (DAP12). This represents the absolute functional extreme of microglial failure; without the TREM2/TYROBP signaling complex, myeloid cells cannot properly mature or survive, leading to catastrophic systemic failure in brain architecture and bone remodeling.

Evidence State

human geneticshuman tissueclinical program

Key Microglial Targets

Last reviewed: June 4, 2026