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Exon-Skipping Oligonucleotides Restore Functional Collagen VI by Correcting a Common COL6A1 Mutation in Ullrich CMD

Crossref DOI link: https://doi.org/10.1016/j.omtn.2020.05.029

Published Print: 2020-09

Update policy: https://doi.org/10.1016/elsevier_cm_policy

Authors

Aguti, Sara

Bolduc, Véronique

Ala, Pierpaolo

Turmaine, Mark

Bönnemann, Carsten G.

Muntoni, Francesco

Zhou, Haiyan
Funding

Funding for this research was provided by:

National Institutes of Health

Great Ormond Street Hospital for Children

National Institute of Neurological Disorders and Stroke

University College London Hospitals NHS Foundation Trust

Wellcome Trust (204841/Z/16/Z)

Muscular Dystrophy UK (17GRO-PG36-0168)

University College London

National Institute for Health and Care Research
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Text and Data Mining valid from 2020-09-01

Text and Data Mining valid from 2020-09-01

Version of Record valid from 2020-05-28
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This article is maintained by: Elsevier

Article Title: Exon-Skipping Oligonucleotides Restore Functional Collagen VI by Correcting a Common COL6A1 Mutation in Ullrich CMD

Journal Title: Molecular Therapy Nucleic Acids

CrossRef DOI link to publisher maintained version: https://doi.org/10.1016/j.omtn.2020.05.029

Content Type: article

Copyright: © 2020 The Author(s).