Publications

Find the latest research articles discussing hematopoietic stem cell transplant (HSCT) using bone marrow for ALSP.

🧠Microglia replacement halts the progression of microgliopathy in mice and humans — by J. Wu et al., 2025

Key Findings: Authors report that replacing CSF1R‑deficient microglia (via transplantation, “tBMT”) halted disease progression over 24 months follow-up in eight human patients (and in animal models).
• This suggests that microglia replacement therapy can correct pathogenic microglial mutations, including those underlying ALSP, and effectively stop or slow neurodegeneration.
• Clinical relevance: this may represent the first “disease‑modifying” therapy for CSF1R‑related leukoencephalopathy / microgliopathy; could shift paradigm from symptomatic/palliative care to curative or stabilizing interventions.