Link between MHC Fiber Type and Restoration of Dystrophin Expression and Key Components of the DAPC by Tricyclo-DNA-Mediated Exon Skipping

Author:

Omairi Saleh,Hau Kwan-Leong,Collin-Hooper Henry,Montanaro Federica,Goyenvalle Aurelie,Garcia Luis,Patel Ketan

Funder

Ministry of Higher Education of Iraq

Agence Nationale de la Recherche

Association Monegasque Contre les Myopathies

Biotechnology and Biological Sciences Research Council (BBSRC-UK)

European Horizon 2020 re-integration Marie-Skłodowska Curie Senior Fellowship

Publisher

Elsevier BV

Subject

Drug Discovery,Molecular Medicine

Reference34 articles.

1. Orphan drug development in muscular dystrophy: update on two large clinical trials of dystrophin rescue therapies;Hoffman;Discov. Med.,2013

2. Twenty-year follow-up of newborn screening for patients with muscular dystrophy;Chung;Muscle Nerve,2016

3. The Duchenne muscular dystrophy gene product is localized in sarcolemma of human skeletal muscle;Zubrzycka-Gaarn;Nature,1988

4. Dystrophin: the protein product of the Duchenne muscular dystrophy locus;Hoffman;Cell,1987

5. Dystrophin-glycoprotein complex is highly enriched in isolated skeletal muscle sarcolemma;Ohlendieck;J. Cell Biol.,1991

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