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Development of an ex vivo precision gene engineered B cell medicine that produces highly active and sustained levels of acid sphingomyelinase for the treatment of Neimann-Pick disease

Monika Musial-Siwek, Ishara Datta, Wayne Bainter, Timothy Mullen, Connor Malloy, Michael Leiken, Amy Lundberg,Sarah Leach, Megan Brophy,Anja Hohmann,Sean Arlauckas,Adam Lazorchak, Claudia Fiorini, Hanlan Liu,Richard A. Morgan

MOLECULAR GENETICS AND METABOLISM（2024）

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