BI-TfR1 CapX rapidly transits the blood-brain barrier for efficient, low-dose gene delivery throughout the CNS
BI-TfR1 CapX is described, a human transferrin receptor (TfR1)-binding AAV capsid that, after a low intravenous dose in adult humanized TFRC mice, transduced more than 80% of cortical and spinal neurons and ∼60% of cortical astrocytes, with reduced distribution and expression in several peripheral tissues relative to A...