A novel siRNA targeting and delivery platform inhibits glycogen synthesis and reduces glycogen levels in skeletal and cardiac muscle in a mouse model of Pompe disease

被引：1
|
作者：
Pederson, Bartholomew A. [1 ]
Holt, Bryce D. [1 ]
Elliot, Samuel J. [1 ]
O'Neil, Karyn [2 ]
Druzina, Zhanna [2 ]
Kulkarni, Swapnil [2 ]
Thurberg, Beth L. [3 ]
Nadler, Steven G. [2 ]
机构：
[1] Ball State Univ, Muncie, IN USA
[2] Aro Biotherapeut, Philadelphia, PA USA
[3] Beth Thurberg Orphan Sci Consulting LLC, Newton, MA USA
来源：
MOLECULAR GENETICS AND METABOLISM | 2024年 / 141卷 / 02期
关键词：
D O I：
10.1016/j.ymgme.2023.107994
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
255
引用
收藏
页数：2
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