Peptide-conjugated PMOs for the treatment of myotonic dystrophy

Peptide-conjugated PMOs for the treatment of myotonic dystrophy

Antisense oligonucleotides (ASOs) have shown great therapeutic potential in the treatment of many neuromuscular diseases including myotonic dystrophy 1 (DM1). However, systemically delivered ASOs display poor biodistribution and display limited penetration into skeletal muscle. The conjugation of ce...

书目详细资料
Main Authors:	Stoodley, J, Miraz, DS, Jad, Y, Fischer, M, Wood, MJA, Varela, MA
其他作者:	Maruyama, R
格式:	Journal article
语言:	English
出版:	Springer 2022

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