PR-009, a new AAV-hTREM2 gene therapy to restore microglial function in patients with ALSP-CSF1R
May 14, 2024
Macrophage colony-stimulating factor 1 receptor (CSF-1R) is a transmembrane tyrosine kinase receptor expressed in brain microglia, and mutations in the CSF1R gene have been linked to adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP).