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

• DOI: 10.1016/j.ymgme.2023.107975
• Publication Date: 2024-01-31T20:44:36Z
• Authors: 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
• Keywords: Acid sphingomyelinase