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BI-hTFR1 was actively transported across human brain endothelial cells and, relative to AAV9, provided 40 to 50 times greater reporter expression in the CNS of human\n                    <jats:italic>TFRC<\/jats:italic>\n                    knockin mice. The enhanced tropism was CNS-specific and absent in wild-type mice. When used to deliver\n                    <jats:italic>GBA1<\/jats:italic>\n                    , mutations of which cause Gaucher disease and are linked to Parkinson\u2019s disease, BI-hTFR1 substantially increased brain and cerebrospinal fluid glucocerebrosidase activity compared with AAV9. 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