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Antisense oligonucleotide targeting glycogen synthase (GYS1) in a Pompe disease mouse model

2020

Kimonis, Virginia E;
Weiss, Lan;
Carrer, Michele;
Ta, Lac;
Chang, Mindy;
Cheng, Cheng;
Hettrick, Lisa;
Watt, Andy;
Raben, Nina;
Grossman, Tamar
et al.

Published Web Location

https://doi.org/10.1016/j.ymgme.2019.11.224

Creative Commons 'BY' version 4.0 license

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