Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice-Reference-Cited by-同舟云学术

Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice

Published:2019-09-30 Issue:11 Volume:129 Page:4739-4744
ISSN:0021-9738
Container-title:Journal of Clinical Investigation
language:en
Short-container-title:

Author:

Klein Arnaud F.^ORCID,Varela Miguel A.,Arandel Ludovic,Holland Ashling^ORCID,Naouar Naira,Arzumanov Andrey,Seoane David,Revillod Lucile,Bassez Guillaume^ORCID,Ferry Arnaud,Jauvin Dominic,Gourdon Genevieve,Puymirat Jack,Gait Michael J.,Furling Denis^ORCID,Wood Matthew J.A.

Funder

Association Française contre les Myopathies

Publisher

American Society for Clinical Investigation

Subject

General Medicine

Link

https://www.jci.org/articles/view/128205/files/pdf

Reference33 articles.

1. Myotonic Dystrophy Mutation: an Unstable CTG Repeat in the 3′ Untranslated region of the Gene

2. Molecular basis of myotonic dystrophy: Expansion of a trinucleotide (CTG) repeat at the 3′ end of a transcript encoding a protein kinase family member

3. An Unstable Triplet Repeat in a Gene Related to Myotonic Muscular Dystrophy

4. Foci of trinucleotide repeat transcripts in nuclei of myotonic dystrophy cells and tissues.

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