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RNAi-mediated Gene Silencing of Mutant Myotilin Improves Myopathy in LGMD1A Mice.
Liu J, Wallace LM, Garwick-Coppens SE, Sloboda DD, Davis CS, Hakim CH, Hauser MA, Brooks SV, Mendell JR, Harper SQ. Liu J, et al. Among authors: harper sq. Mol Ther Nucleic Acids. 2014 Apr 29;3(4):e160. doi: 10.1038/mtna.2014.13. Mol Ther Nucleic Acids. 2014. PMID: 24781192 Free PMC article.
Aberrant splicing in transgenes containing introns, exons, and V5 epitopes: lessons from developing an FSHD mouse model expressing a D4Z4 repeat with flanking genomic sequences.
Ansseau E, Domire JS, Wallace LM, Eidahl JO, Guckes SM, Giesige CR, Pyne NK, Belayew A, Harper SQ. Ansseau E, et al. Among authors: harper sq. PLoS One. 2015 Mar 5;10(3):e0118813. doi: 10.1371/journal.pone.0118813. eCollection 2015. PLoS One. 2015. PMID: 25742305 Free PMC article.
55 results