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4. Networking to Optimize Dmd exon 53 Skipping in the Brain of mdx52 Mouse Model

Author

Doisy, Mathilde, primary, Vacca, Ophélie, additional, Fergus, Claire, additional, Gileadi, Talia, additional, Verhaeg, Minou, additional, Saoudi, Amel, additional, Tensorer, Thomas, additional, Garcia, Luis, additional, Kelly, Vincent P., additional, Montanaro, Federica, additional, Morgan, Jennifer E., additional, van Putten, Maaike, additional, Aartsma-Rus, Annemieke, additional, Vaillend, Cyrille, additional, Muntoni, Francesco, additional, and Goyenvalle, Aurélie, additional

Published
2023
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6. Oligonucleotide Enhancing Compound Increases Tricyclo-DNA Mediated Exon-Skipping Efficacy in the Mdx Mouse Model

Author

Bizot, Flavien, primary, Fayssoil, Abdallah, additional, Gastaldi, Cécile, additional, Irawan, Tabitha, additional, Phongsavanh, Xaysongkhame, additional, Mansart, Arnaud, additional, Tensorer, Thomas, additional, Brisebard, Elise, additional, Garcia, Luis, additional, Juliano, Rudolph L, additional, and Goyenvalle, Aurélie, additional

Published
2023
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7. Dystrophin myonuclear domain restoration governs treatment efficacy in dystrophic muscle

Author

Morin, Adrien, primary, Stantzou, Amalia, additional, Petrova, Olga N., additional, Hildyard, John, additional, Tensorer, Thomas, additional, Matouk, Meriem, additional, Petkova, Mina V., additional, Richard, Isabelle, additional, Manoliu, Tudor, additional, Goyenvalle, Aurélie, additional, Falcone, Sestina, additional, Schuelke, Markus, additional, Laplace-Builhé, Corinne, additional, Piercy, Richard J., additional, Garcia, Luis, additional, and Amthor, Helge, additional

Published
2023
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8. Palmitic acid conjugation enhances potency of tricyclo-DNA splice switching oligonucleotides

Author

Relizani, Karima, primary, Echevarría, Lucía, additional, Zarrouki, Faouzi, additional, Gastaldi, Cécile, additional, Dambrune, Chloe, additional, Aupy, Philippine, additional, Haeberli, Adrian, additional, Komisarski, Marek, additional, Tensorer, Thomas, additional, Larcher, Thibaut, additional, Svinartchouk, Fedor, additional, Vaillend, Cyrille, additional, Garcia, Luis, additional, and Goyenvalle, Aurélie, additional

Published
2021
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9. Dystrophin myonuclear domain restoration governs treatment efficacy in dystrophic muscle.

Author

Morina, Adrien, Stantzou, Amalia, Petrova, Olga N., Hildyard, John, Tensorer, Thomas, Matouk, Meriem, Petkova, Mina V., Richard, Isabelle, Manoliu, Tudor, Goyenvallea, Aurélie, Falcone, Sestina, Schuelke, Markus, Laplace-Builhé, Corinne, Piercy, Richard J., Garcia, Luis, and Amthor, Helge

Subjects
DYSTROPHIN, TREATMENT effectiveness, DUCHENNE muscular dystrophy, MUSCULAR dystrophy, GENOME editing
Abstract

Dystrophin is essential for muscle health: its sarcolemmal absence causes the fatal, X-linked condition, Duchenne muscular dystrophy (DMD). However, its normal, spatial organization remains poorly understood, which hinders the interpretation of efficacy of its therapeutic restoration. Using female reporter mice heterozygous for fluorescently tagged dystrophin (DmdEGFP), we here reveal that dystrophin distribution is unexpectedly compartmentalized, being restricted to myonuclear-defined sarcolemmal territories extending ~80 µm, which we called “basal sarcolemmal dystrophin units (BSDUs).” These territories were further specialized at myotendinous junctions, where both Dmd transcripts and dystrophin protein were enriched. Genome-level correction in X-linked muscular dystrophy mice via CRISPR/Cas9 gene editing restored a mosaic of separated dystrophin domains, whereas transcript-level Dmd correction, following treatment with tricyclo-DNA antisense oligonucleotides, restored dystrophin initially at junctions before extending along the entire fiber—with levels ~2% sufficient to moderate the dystrophic process. We conclude that widespread restoration of fiber dystrophin is likely critical for therapeutic success in DMD, perhaps most importantly, at muscle–tendon junctions. [ABSTRACT FROM AUTHOR]

Published
2023
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