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    INNOVATIVE SPLICE SWITCHING OLIGONUCLEOTIDE THERAPY FOR NIEMANN-PICK TYPE C1 DISEASE

    INNOVATIVE SPLICE SWITCHING OLIGONUCLEOTIDE THERAPY FOR NIEMANN-PICK TYPE C1 DISEASE by Maria Martinez De Lagran, Yu-Han Huang, Ondrej Kostov, Haiyan Zhou, Paul Gissen, Marvin Caruthers, Timothy Yu, Daniel Ginberg, Mara Dierssen

    Published 2023-10-01
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