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  1. 1
    Modulation of mTOR signaling as a strategy for the treatment of Pompe disease

    Modulation of mTOR signaling as a strategy for the treatment of Pompe disease by Jeong‐A Lim, Lishu Li, Orian S Shirihai, Kyle M Trudeau, Rosa Puertollano, Nina Raben

    Published 2017-01-01
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  2. 2
    Skeletal muscle effects of antisense oligonucleotides targeting glycogen synthase 1 in a mouse model of Pompe disease

    Skeletal muscle effects of antisense oligonucleotides targeting glycogen synthase 1 in a mouse model of Pompe disease by Lan Weiss, Michele Carrer, Alyaa Shmara, Angela Martin, Hong Yin, Pallabi Pal, Cheng Cheng, Lac Ta, Victoria Boock, Yasamin Fazeli, Mindy Chang, Marvin Paguio, Jonathan Lee, Howard Yu, John Weiss, Tamar R Grossman, Nina Raben, Paymaan Jafar‐Nejad, Virginia Kimonis

    Published 2025-04-01
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