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. 2020 Dec 10;11:594576. doi: 10.3389/fgene.2020.594576

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Copyright © 2020 Zhang, Bewick, Xia, Furling and Ashizawa.

This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

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LshCas13a reverses downstream missplicing events and knocks down DMPK mRNA quantity in transduced DM1 myoblasts. (A) Splicing patterns from six MBNL1-regulated genes were analyzed in normal myoblasts, untreated DM1 myoblasts, DM1 myoblasts NT and DM1 myoblasts targeting. The ratios of top and bottom bands (as indicated by arrows) were quantified with standard deviation (±SD) in Fiji ImagJ from two separate experiments. (B) The DMPK mRNA level was measured using qRT-PCR from the four cell lines. The relative DMPK mRNA level from each cell line was normalized to that of the normal myoblast (white bar). Three biological replicates each with three technical replicates were used in this assay.