Enhanced exon skipping and prolonged dystrophin restoration achieved by TfR1-targeted delivery of antisense oligonucleotide using FORCE conjugation in mdx mice
Author(s) -
Cody A. Desjardins,
Monica Yao,
John K. Hall,
Emma O’Donnell,
Reshmii Venkatesan,
Sean Spring,
Aiyun Wen,
Nelson Hsia,
Peiyi Shen,
Ryan Russo,
Bo Lan,
Tyler Picariello,
Kim San Tang,
Timothy Weeden,
Stefano Zanotti,
Romesh R. Subramanian,
Oxana IbraghimovBeskrovnaya
Publication year - 2022
Publication title -
nucleic acids research
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 9.008
H-Index - 537
eISSN - 1362-4954
pISSN - 0305-1048
DOI - 10.1093/nar/gkac641
Subject(s) - dystrophin , duchenne muscular dystrophy , morpholino , exon skipping , exon , mdx mouse , biology , skeletal muscle , sarcolemma , utrophin , microbiology and biotechnology , endocrinology , biochemistry , genetics , alternative splicing , gene knockdown , gene
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