Antisense oligonucleotides in myotonic dystrophy type 1: lessons learnt
Antisense oligonucleotides in myotonic dystrophy type 1: lessons learnt
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Publisher
England: Elsevier Ltd
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Language
English
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Publisher
England: Elsevier Ltd
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Scope and Contents
Contents
[...]the findings of this trial highlight that the reasons behind the limited use of ASOs in clinical and research settings—namely inadequate target engagement and insufficient biological activity—still held true. Nonetheless, Thornton and colleagues did a post-hoc targeted RNA sequencing analysis using a composite splicing index from patients trea...
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Full title
Antisense oligonucleotides in myotonic dystrophy type 1: lessons learnt
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TN_cdi_proquest_miscellaneous_2778973064
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_2778973064
Other Identifiers
ISSN
1474-4422
E-ISSN
1474-4465
DOI
10.1016/S1474-4422(23)00040-6
