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Novel FACS Based Method Demonstrates CNS Cell-Type Distribution and Efficacy of a BBB Penetrant ERT in a Mouse Model of MPS II

Julie C. Ullman,Alicia A. Nugent,Annie Arguello,Akhil Bhalla,Thomas Sandmann,Steve Lianoglou,Connie Ha,Junhua Wang,Sonnet S. Davis,Jamal Alkabsh,Giuseppe Astarita,Gilbert Di Paolo,Tina Giese,Cathal Mahon,Catherine Bedard,Nicholas Liang,Gunasekaran Kannan, Joesph W. Lewcock,Zachary K. Sweeney,Mihalis S. Kariolis,Dolo Diaz,Anastasia Henry

MOLECULAR GENETICS AND METABOLISM（2020）

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