From Cryptic Toward Canonical Pre-mRNA Splicing in Pompe Disease: a Pipeline for the Development of Antisense Oligonucleotides

Published 2016 View Full Article

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Title
From Cryptic Toward Canonical Pre-mRNA Splicing in Pompe Disease: a Pipeline for the Development of Antisense Oligonucleotides
Authors
Keywords
cryptic splice site, morpholino antisense oligonucleotides, muscle, Pompe disease, splicing correction
Journal
Molecular Therapy-Nucleic Acids
Volume 5, Issue -, Pages e361
Publisher
Elsevier BV
Online
2016-09-13
DOI
10.1038/mtna.2016.75

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